StemCell Therapy

DGAP-News: MorphoSys AG / Key word(s): Miscellaneous04.06.2021 / 19:32 The issuer is solely responsible for the content of this announcement.

Media Release

MorphoSys and Incyte Announce Three-Year Results from Phase 2 L-MIND Study of Tafasitamab in Combination with Lenalidomide for the Treatment of Relapsed or Refractory DLBCL

Presentation will be available on demand as part of the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting

BOSTON, Mass., USA and WILMINGTON, Del., USA - June 4, 2021 - MorphoSys US Inc., a fully owned subsidiary of MorphoSys AG (FSE: MOR; NASDAQ: MOR), and Incyte (NASDAQ:INCY) today announced new three-year follow-up data from the ongoing Phase 2 L-MIND study of tafasitamab (Monjuvi(R)) in combination with lenalidomide in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). A total of 80 out of 81 enrolled study patients receiving tafasitamab plus lenalidomide were included in the efficacy analysis at approximately three years follow-up (>=35 months).[1] The long-term analysis, as assessed by an independent review committee (IRC), showed that patients treated with tafasitamab plus lenalidomide had an overall response rate (ORR) of 57.5% (95% CI = 45.9%, 68.5%; 46 out of 80 patients), including a complete response (CR) rate of 40% (32 out of 80 patients). Additionally, the median duration of response (DoR) was 43.9 months (95% CI = 26.1, NR), with a median overall survival (OS) of 33.5 months (95% CI = 18.3, NR) and median progression free survival (PFS) of 11.6 months (95% CI = 6.3, 45.7).

These data (abstract #7513) are available on demand as part of the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, held virtually June 4-8, 2021, and will be presented as a poster and poster discussion in the Hematologic Malignancies-Lymphoma and Chronic Lymphocytic Leukemia session.

"The three-year efficacy data, combined with the safety and tolerability profile of tafasitamab, further support a therapeutic option for patients with relapsed or refractory DLBCL who are ineligible for transplant - a traditionally difficult-to-treat population," said Gilles Salles, M.D., Ph.D., Lymphoma Service Chief at Memorial Sloan Kettering Cancer Center, and lead investigator of the L-MIND study*. "I am encouraged to see the confirmed favorable outcome of patients in the L-MIND study, which suggest that this combination treatment regimen could potentially offer a paradigm shift and long-term disease control."

The new results - based on an October 30, 2020 data cut-off - build on previous findings showing durable responses and a consistent safety profile of tafasitamab in combination with lenalidomide followed by tafasitamab monotherapy in autologous stem cell transplantation (ASCT)-ineligible patients with relapsed or refractory DLBCL.

"The three-year follow-up data not only show a durable response and consistent safety profile in patients with relapsed or refractory DLBCL treated with tafasitamab plus lenalidomide, it also suggests the combination could potentially lead to durable remission," said Nuwan Kurukulasuriya, Ph.D., Senior Vice President, Global Head of Medical Affairs, MorphoSys. "We are looking forward to sharing these long-term follow-up findings with the scientific community."

"We are pleased that long-term data from the L-MIND study underscore the clinically-significant durable responses that are possible with tafasitamab plus lenalidomide as a treatment for relapsed or refractory DLBCL," said Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapies, Incyte. "We look forward to continuing to build the body of clinical evidence supporting tafasitamab as a treatment option for patients with DLBCL, as well as exploring other potential indications for tafasitamab through our ongoing research and development program."

In July 2020, the U.S. Food and Drug Administration (FDA) approved Monjuvi(R) (tafasitamab-cxix) in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for ASCT. This indication is approved under accelerated approval based on ORR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). The U.S. approval is based on an efficacy subgroup of 71 patients confirmed by central lab.

The FDA decision represented the first approval of a second-line treatment for adult patients with DLBCL who progressed during or after first-line therapy.

About Diffuse Large B-cell Lymphoma (DLBCL)DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide[2], characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs. It is an aggressive disease with about 40% of patients not responding to initial therapy or relapsing thereafter[3], leading to a high medical need for new, effective therapies[4], especially for patients who are not eligible for an autologous stem cell transplant in this setting.

About L-MINDThe L-MIND trial is a single arm, open-label Phase 2 study (NCT02399085) investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who have had at least one, but no more than three prior lines of therapy, including an anti-CD20 targeting therapy (e.g., rituximab), who are not eligible for high-dose chemotherapy or refuse subsequent autologous stem cell transplant. The study's primary endpoint is overall response rate (ORR). Secondary outcome measures include duration of response (DoR), progression-free survival (PFS) and overall survival (OS). In May 2019, the study reached its primary completion.

For more information about L-MIND, visit https://clinicaltrials.gov/ct2/show/NCT02399085.

About TafasitamabTafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi(R) is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the European Union has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi(R) is a registered trademark of MorphoSys AG.

XmAb(R) is a registered trademark of Xencor, Inc.

Important Safety Information

What are the possible side effects of MONJUVI?

MONJUVI may cause serious side effects, including:

- Infusion reactions. Your healthcare provider will monitor you for infusion reactions during your infusion of MONJUVI. Tell your healthcare provider right away if you get fever, chills, rash, flushing, headache, or shortness of breath during an infusion of MONJUVI.

- Low blood cell counts (platelets, red blood cells, and white blood cells). Low blood cell counts are common with MONJUVI, but can also be serious or severe. Your healthcare provider will monitor your blood counts during treatment with MONJUVI. Tell your healthcare provider right away if you get a fever of 100.4 F (38 C) or above, or any bruising or bleeding.

- Infections. Serious infections, including infections that can cause death, have happened in people during treatments with MONJUVI and after the last dose. Tell your healthcare provider right away if you get a fever of 100.4 F (38 C) or above, or develop any signs and symptoms of an infection.

The most common side effects of MONJUVI include:

- Feeling tired or weak

- Diarrhea

- Cough

- Fever

- Swelling of lower legs or hands

- Respiratory tract infection

- Decreased appetite

These are not all the possible side effects of MONJUVI.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you:

- Have an active infection or have had one recently.

- Are pregnant or plan to become pregnant. MONJUVI may harm your unborn baby. You should not become pregnant during treatment with MONJUVI. Do not receive treatment with MONJUVI in combination with lenalidomide if you are pregnant because lenalidomide can cause birth defects and death of your unborn baby.

- You should use an effective method of birth control (contraception) during treatment and for at least 3 months after your final dose of MONJUVI.

- Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with MONJUVI.

- Are breastfeeding or plan to breastfeed. It is not known if MONJUVI passes into your breastmilk. Do not breastfeed during treatment for at least 3 months after your last dose of MONJUVI.

You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

Tell your healthcare provider about all the medications you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Please see the full Prescribing Information for Monjuvi, including Patient Information, for additional Important Safety Information.

About MorphoSysMorphoSys (FSE & NASDAQ: MOR) is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for people living with cancer and autoimmune diseases. Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys is advancing its own pipeline of new drug candidates and has created antibodies which are developed by partners in different areas of unmet medical need. In 2017, Tremfya(R) (guselkumab) - developed by Janssen Research & Development, LLC and marketed by Janssen Biotech, Inc., for the treatment of plaque psoriasis - became the first drug based on MorphoSys' antibody technology to receive regulatory approval. In July 2020, the U.S. Food and Drug Administration (FDA) granted accelerated approval of the company's proprietary product Monjuvi(R) (tafasitamab-cxix) in combination with lenalidomide in patients with a certain type of lymphoma. Headquartered near Munich, Germany, the MorphoSys group, including the fully owned U.S. subsidiary MorphoSys US Inc., has more than 600 employees. More information at http://www.morphosys.com or http://www.morphosys-us.com.

Monjuvi(R) is a registered trademark of MorphoSys AG.

Tremfya(R) is a registered trademark of Janssen Biotech, Inc.

About Incyte Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

MorphoSys Forward-looking Statements This communication contains certain forward-looking statements concerning the MorphoSys group of companies, including the expectations regarding Monjuvi's ability to treat patients with relapsed or refractory diffuse large B-cell lymphoma, the further clinical development of tafasitamab-cxix, including ongoing confirmatory trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings and possible additional approvals for tafasitamab-cxix as well as the commercial performance of Monjuvi. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "would," "could," "potential," "possible," "hope" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The forward-looking statements contained herein represent the judgment of MorphoSys as of the date of this release and involve known and unknown risks and uncertainties, which might cause the actual results, financial condition and liquidity, performance or achievements of MorphoSys, or industry results, to be materially different from any historic or future results, financial conditions and liquidity, performance or achievements expressed or implied by such forward-looking statements. In addition, even if MorphoSys' results, performance, financial condition and liquidity, and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. Among the factors that may result in differences are MorphoSys' expectations regarding risks and uncertainties related to the impact of the COVID-19 pandemic to MorphoSys' business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products, the global collaboration and license agreement for tafasitamab, the further clinical development of tafasitamab, including ongoing confirmatory trials, and MorphoSys' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings and possible additional approvals for tafasitamab-cxix as well as the commercial performance of Monjuvi, MorphoSys' reliance on collaborations with third parties, estimating the commercial potential of its development programs and other risks indicated in the risk factors included in MorphoSys' Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. MorphoSys expressly disclaims any obligation to update any such forward-looking statements in this document to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements, unless specifically required by law or regulation.

Incyte Forward-looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the Company's expectations regarding the use of tafasitamab for treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), its ongoing clinical development program for tafasitamab, its L-MIND program, its diffuse large B-cell lymphoma (DLBCL) program generally and its further discussions with regulators regarding tafasitamab as a treatment for patients with DLBCL or for any other indication, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; the effects of the COVID-19 pandemic and measures to address the pandemic on the Company's clinical trials, supply chain, and other third-party providers and development and discovery operations; determinations made by the FDA, European Medicines Agency (EMA), or other regulatory authorities; the Company's dependence on its relationships with its collaboration partners; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission, including its annual report and its quarterly report on Form 10-Q for the quarter ended March 31, 2021. The Company disclaims any intent or obligation to update these forward-looking statements.

*Dr. Salles has provided speaking and advisory services to MorphoSys and Incyte.

Contacts:

[1] Johannes Duell, M.D., et al. Long-term analyses from L-MIND, a Phase II study of tafasitamab (MOR208) combined with lenalidomide (LEN) in patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). 2021 American Society of Clinical Oncology (ASCO) Annual Meeting. Abstract #7513.[2] Sarkozy C, et al. Management of relapsed/refractory DLBCL. Best Practice Research & Clinical Haematology. 2018 31:209-16. doi.org/10.1016/j.beha.2018.07.014.[3] Skrabek P, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Current Oncology. 2019 26(4): 253-265. doi.org/10.3747/co.26.5421.[4] Skrabek P, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Current Oncology. 2019 26(4): 253-265. doi.org/10.3747/co.26.5421.

04.06.2021 Dissemination of a Corporate News, transmitted by DGAP - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.

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MorphoSys and Incyte Announce Three-Year Results from Phase 2 L-MIND Study of Tafasitamab in Combination with Lenalidomide for the Treatment of...

"Cancer is a disease of genetics, yet clinical practice has struggled to keep pace with rapid advancements in research, particularly with respect to the role of germline genetics. Testing guidelines and medical policy often codify barriers, further lengthening the path to adoption of widespread testing and in some cases restricting access to precision therapies and clinical treatment trials," said Ed Esplin, M.D., Ph.D., FACMG, FACP, clinical geneticist at Invitae. "Research presented at ASCO shows that cancer-linked genetic changes are common across cancer types and when patients do receive germline testing, over two thirds of those with positive results are eligible for changes to their treatment plans. It's clear that incorporating germline testing alongside tumor profiling can help oncologists better tailor treatment for each patient."

Data from 250 pancreatic cancer patients from the landmark INTERCEPT study conducted at the Mayo Clinic found that nearly one in six patients with pancreatic cancer (n=38) showed cancer-linked genetic changes and, importantly, receiving germline testing was associated with improved survival.

A separate study of prostate cancer patients confirmed similar findings in other cancer types that limiting testing deprives patients and clinicians of actionable information. In the first-ever presentation of the PROCLAIM study, which was conducted primarily in community urology clinics, of patients diagnosed with prostate cancer, a significant number of cancer-linked variants were missed if testing was done based on NCCN guidelines. Of the 532 patients with clinician-reported data, nearly half, 45% (n=239), did not meet NCCN criteria. Overall, 59 patients had a cancer-linked variant; one in 10 of them did not meet the criteria (9.6%, n=23), and 12.3% (n=36) of patients met the criteria. When a 12-gene panel was used, only 29 patients were found to have a cancer-linked variant and one third of these patients were missed by guidelines.

A third study showed simply changing medical policy is not enough to drive changes in clinician adoption. In a review of two independent datasets, including commercially insured and Medicare Advantage enrollees, only 3% (n=1,675) of the 55,595 colorectal cancer patients received germline genetic testing, despite medical policy recommending germline genetic testing for all colorectal cancer patients (consistent with the INTERCEPT colorectal cancer study). Of the patients who received testing, 18% (n=143) had a cancer-linked variant and two thirds, or 67% (n=96), of those patients were potentially eligible for precision therapy and/or clinical trials.

"The data have been available for years that show knowing what changes patients have in their genes is beneficial to treating their cancer. Yet the oncology community has been slower to adopt germline testing than tumor profiling, for reasons that are not entirely clear. These data presented at ASCO highlight the need for oncologists to embrace germline genetic testing as routine practice for all cancer patients," said Robert Nussbaum, M.D., chief medical officer at Invitae. "A positive germline genetic result may enable patients to enroll in clinical trials or gain access to new precision medicines. And equally important, the discovery of an inherited variant can alert relatives to seek out earlier cancer screening, helping avoid later-stage diagnoses and offering a treatment benefit if cancer develops."

Invitae aims to help overcome obstacles to the adoption of genetic testing by providing physicians with clinical consults to help interpret results and reducing cost as a barrier to genetic information. Invitae also provides patients direct access to genetic counselors, helping to integrate routine genetic testing into patient care with GIA, a HIPAA-compliant chatbot. Family members are also able to receive no-charge genetic testing if a positive result is found.

Details of the 2021 ASCO presentations:

Oral Abstract Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Discussion Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Gastrointestinal Cancer--GastroesophageaI, Pancreatic, and Hepatobiliary

About InvitaeInvitae Corporation(NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Safe Harbor StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the benefits of germline testing and genetic information; and that the data presented at ASCO highlight the need for increased germline testing in all cancer patients regardless of medical policy. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact:Laura D'Angelo[emailprotected](628) 213-3283

SOURCE Invitae Corporation

http://www.invitae.com

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Germline genetic testing can benefit all cancer patients as a routine practice in cancer care - PRNewswire

The loss of diversity in fields and gardens has real consequences, according to Tommy Carter, USDA professor, soybean breeding and genetics emeritus faculty, North Carolina State University. Carter explains the importance of increasing genetic diversity in crops in this Sustainable, Secure Food blog.

Modern crop varieties are often too uniform genetically speaking for good agricultural health, Carter writes. Thats because many new varieties are too closely related like cousins or siblings. This uniformity makes them less useful as breeding stock in current breeding efforts because they have lost useful genes which are still present in the landraces.

Soybean provides a good example regarding the insufficient diversity in modern varieties. Farmers domesticated soybean perhaps five thousand years ago in central China. These seeds spread through most of Asia via caravans with population migration. Adapting soybean to local conditions as soybean spread slowly over Asia, ancient farmers selected out more than 10,000 diverse varieties from domestication to the present. Many of these are now preserved by USDA and China in seed banks.

Although the thousands of old Asian soybean landraces are genetically diverse, modern U.S varieties are not. In the process of developing modern soybean varieties for U.S. farmers, the first generations of U.S. soybean breeders (~1930-1990) essentially ignored genetic diversity. They instead focused on adapting soybean for mechanical farming. Hundreds of new varieties were released to U.S. farmers in a successful endeavor to improve productivity, but these varieties were not very diverse, genetically speaking.

Today, U.S. soybean breeding programs are widely recognized as limited by insufficient genetic diversity. Breeding progress slowed, and the reasons are twofold:

Two landmark soybean USDA cultivars, Lee and Forrest, in the southern U.S. offer prime examples of this problem. They were released in the 1950s and 1970s. Their superior agronomics and popularity on the farm led to their heavy use as parental stocks for breeding during the following decades.

The result was a new generation of progeny (soybean children) that were highly related not only to the landmark varieties Lee and Forrest, but to each other as well. Although they performed well in the field, these brother and sister soybeans were not good mating stock for producing new varieties. The term inbreeding is often used to describe this effect in animal breeding, and the term applies here as well.

Short-term gains made in developing Lee and Forrest, thus, came at the expense of long-term progress. Diversity, the basis for new progress, was lost. But a new plan from the USDA-ARS, known as the 301 Plan, has the goal to restore diversity to applied breeding programs. Science in the 301 Plan results in new, unique breeding lines which have diverse pedigrees and genetics.

A new release of soybean USDA-N6004 is part of that effort. When new varieties of plants are certified by the USDA, they receive an official registration number. Some breeders then choose to name their variety with a more common name, such as Lee and Forrest soybean mentioned. Breeders created USDA-N6004 soybean by hybridizing of USDA cultivar NC-Roy and Japanese cultivar Blue Side. Blue Side is a vegetable (edamame) soybean that comes from outside the U.S.s genetic base. Japanese germplasm generally is not well represented as parental stock in U.S. breeding. Thus, Japan appears to be a rich untapped source of diverse genes for future U.S. soybean breeding.

Source: Sustainable, Secure Food blog written by members of the American Society of Agronomy and Crop Science Society of America, which is solely responsible for the information provided and is wholly owned by the source. Informa Business Media and all its subsidiaries are not responsible for any of the content contained in this information asset.

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Increasing genetic diversity in crops is important - Farm Progress

Scientists at the Clemson University Center for Human Genetics used single cell transcriptomics technology to identify how acute cocaine exposure affects specific cell clusters in the brain of the common fruit fly, Drosophila melanogaster. The studies, headed by geneticists Trudy Mackay, PhD, and Robert Anholt, PhD, found that cocaine use by the fruit flies elicited rapid, widespread changes in gene expression throughout the brain, and that the differences were more pronounced in males than in females. The investigators hope that the resulting atlas of sexually dimorphic cocaine-modulated gene expression could potentially lay the groundwork for developing drugs that would treat or prevent cocaine addiction in humans.

This research identifies the regions of the brain which are important, said Mackay, the Self Family Endowed Chair in Human Genetics. Now, we can see what genes are expressed when exposed to cocaine and whether there are Federal Drug Administration-approved drugs that could be tested, perhaps first in the fly model. Weve already spotted several of these genes. This is a baseline. We can now leverage this work to understand potential therapy.

Mackay, Anholt and colleagues report on their findings in Genome Research, in a paper titled, The Drosophila brain on cocaine at single cell resolution.

The propensity for cocaine use depends on both genetic and environmental factors, making it hard to study. And while the neurological effects of the drug are well known, scientists know much less about how gene variation may impact on sensitivity to the drugs effects. Furthermore, little is known about acute effects of cocaine consumption on genome-wide gene expression across the brain, they continued.

The fruit fly Drosophila melanogaster is a useful model for systems genetic analysis of cocaine consumption. The majority of the fruit fly genes have human counterparts, providing researchers with a comparable model when studying complex genetic traits. Flies can be reared rapidly in large numbers at low cost in defined genetic backgrounds and under controlled environmental conditions, and about 75% of disease-causing genes in humans have fly orthologs, the team pointed out.

Fruit flies exposed to cocaine showed impaired locomotor activity and increased seizures and, as the authors explained, exposure to cocaine also elicits motor responses in the fruit fly that resemble behaviors observed in rodents. flies in addition develop sensitization to repeated intermittent exposure to cocaine.

For their reported studies, the investigators allowed male and female flies to ingest a fixed amount of sucrose or sucrose supplemented with cocaine, over no more than two hours. Observation of the flies behavior after cocaine ingestion showed evidence that the drug exposure resulted in physiological and behavioral effects, including seizures and compulsive grooming.

To assess the effects of cocaine consumption on gene expression in the brain, the researchers dissected the fly brains into single cells. Using next-generation RNA sequencing technology they were able to make libraries of the expressed genes for individual cells.

To identify specific cell populations that respond to acute cocaine exposure, we analyzed single cell transcriptional responses in duplicate samples of flies that consumed fixed amounts of sucrose or sucrose supplemented with cocaine, in both sexes, the scientists explained. The single-cell technique is ultra-powerful and offers advantages over standard gene expression profile studies. If an entire brain is used and theres heterogeneity of gene expression, such that its up in one cell and down in another, you dont see any signal, Mackay commented. But with the single cell analysis, were able to capture those very, very fine details that reflect heterogeneity in gene expression among different cell types. It is very exciting to apply this advanced technology here at the CHG.

The investigators looked at 88,991 cells, with each cell having thousands of transcripts. Through sophisticated statistical analysis, the researchers were able to categorize the cells into 36 distinct cell clusters. Annotation of clusters based on their gene markers revealed that all major cell typesneuronal and glialas well as neurotransmitter types from most brain regions, including mushroom bodies, were represented. The study results showed that all types of fly brain cells were affected, especially Kenyon cells in the fly brains mushroom bodies, and some glia cells. Mushroom bodies, which get their name because they look like a pair of mushrooms, are integrative brain centers that are associated with experience-dependent behavioral modifications.

Interestingly, the study highlighted extensive sexual dimorphism in the response to cocaine. We found the effects of cocaine in the brain are very widespread, and there are distinct differences between males and females, added Anholt, Provosts Distinguished Professor of Genetics and Biochemistry. The investigators further stated, although cocaine-modulated changes in gene expression are widespread throughout the brain in both sexes, specific changes in transcript abundances are distinct between males and females We identified 691 differentially expressed genes in males and 322 in females following acute exposure to cocaine, of which ~69% have human orthologs. The scientists say the observed sexual dimorphism is in line with previous studies that showed reduced locomotion and increased grooming in flies given low doses of cocaine, with males showing more profound effects than femails.

The collective results of the teams analyses were used to generate what they say is an atlas of sexually dimorphic cocaine-modulated gene expression in a model brain. Ahnolt said its hoped that the atlas will serve as a resource for the research community.

functional parallels between the fly model and human studies provide proof of principle that results from cocaine exposure obtained from the fly model can be translated to human populations, the investigators stated. Thus, the comprehensive documentation of cocaine mediated modulation of gene expression which we have derived can serve as a contextual framework for future human studies.

Mackay is one of the worlds leading authorities on the genetics of complex traits. She has a longstanding interest in behavioral genetics and developing the fruit fly as a model for understanding the genetic basis of complex behaviors. Her laboratory developed the Drosophila melanogaster Genetic Reference Panel (DGRP), which now consists of 1,000 inbred fly lines with fully sequenced genomes derived from a natural population. The DGRP allows researchers to use naturally occurring variation to examine genetic variants that contribute to susceptibility to various stressors.

Excerpt from:
Atlas of Cocaines Effects on Gene Expression Mapped at Single-Cell Level in Fruit Fly Brain - Genetic Engineering & Biotechnology News

The consortium has been exploring the genetics that determine whether fish are resistant to Flavobacterium psychrophilum a bacterium which can lead to health issues in salmon fry.

The discovery is expected to pave the way for selective breeding programmes, which could boost the health and welfare of farmed Scottish salmon by breeding new fish from parents that possess the genetic resistance markers and are, therefore, expected to display increased resistance to the bacteria.

Flavobacteriosis can be a particular threat to smaller, juvenile fish and is a widespread challenge for the aquaculture sector, with infections also reported in Chile, Norway and Canada. However, current prevention and treatment programmes are limited vaccination by injection cannot be used due to the size of the fish and, as the sector continues to move away from antibiotic treatments, a genetic breakthrough could hold the key.

The project is backed by the Sustainable Aquaculture Innovation Centre (SAIC) and led by AquaGen Scotland, with partners from the University of Stirlings Institute of Aquaculture, DawnFresh Farming and Cooke Aquaculture Scotland.

The Health and Welfare of Atlantic Salmon course

It is vital that fish farm operatives who are responsible for farmed fish are trained in their health andwelfare. This will help to ensure that fish are free from disease and suffering whilst at the same timepromote good productivity and comply with legislation.

Andrew Reeve, managing director of AquaGen, said: Continual improvements in fish health and welfare are priorities for the aquaculture industry, to which robust stock suited to the farmed environment make an important contribution. Genetic markers for disease resistance, such as those discovered through this SAIC-funded project, are valuable tools that can and will be immediately employed in breeding work.

To identify the two genetic markers, more than 4,000 fish from AquaGen were tested for more than 70,000 genetic markers using a specially designed lab-based model, which mimics the natural infection route. The next stage of the research programme is to conduct field trials at one of Cooke Aquacultures sites, using salmon eggs specifically selected by AquaGen. It is hoped that, in the event of a natural outbreak of the bacterial disease being detected, these fish can be tested to validate the effect of the genetic markers.

Heather Jones, CEO of SAIC, said: The interim results of this R&D project are highly encouraging and point towards a new, sustainable approach to tackling a common health issue reported in young salmon. One of the most valuable outputs of collaborative innovation projects is the wealth of knowledge that can be shared across the entire sector and findings like this have the power to make a big difference to fish health and welfare.

Dr Rowena Hoare, research fellow at the Institute of Aquaculture, added: Flavobacteriosis is known to be problematic for salmonid culture in freshwater globally for decades. This project has shown how fruitful it can be to combine the expertise of academic and industry researchers to address a complex and economically important disease.

Read more:
Genetic breakthrough could save farmed salmon from flavobacteriosis - The Fish Site

The story of human history is one of migrations over the globe and admixture the exchange of DNA between populations.

Two of the most dramatic of these migrations were slavery and European colonisation. The subsequent admixture between slaves, Europeans and indigenous populations led to the formation of new populations. One, at the southern tip of Africa, was a group that became known as Afrikaners.

Afrikaners predominantly stem from Dutch, French and German immigrants who settled in the Cape, in South Africa, during the second half of the 17th century and the first half of the 18th. Although later European immigrants were also absorbed into the population, their genetic contribution was comparatively small. Another small but significant genetic contribution came from slaves and the local, indigenous Khoekhoe and San populations. These groups were, respectively, pastoralists and hunter-gatherers and in this article we refer to them as the Khoe-San.

Ironically, despite Afrikaners admixed roots, they rose to notoriety for their draconian laws that aimed to segregate groups of people apartheid to allow discrimination against those not of European descent.

The colonisers required labourers and turned to slavery. In fact, there were more slaves than colonists at the Cape during the century preceding the abolition of the slave trade in 1807. The first 400 of these slaves arrived from West Africa in 1658. An estimated 63,000 slaves followed during the next 149 years. During the 17th and 18th centuries, most slaves came from South Asia. Slaves forcefully relocated to the Cape at the end of the 18th century predominantly came from East Africa.

People are, naturally, fascinated by their history. However, it is often poorly documented, recorded with bias, or not recorded at all. Given the central role that ethnicity played and still plays in South African politics, it would be good to have an unbiased estimate of Afrikaners genetic history. We set out to learn more about admixture in the formation of Afrikaners by looking at the genetic variation in their genomes.

Our research had six main findings. First, it confirmed the timing of admixture in the Cape. Second, it showed limited genetic contribution from southern Bantu-speakers, African farmers that colonised southern Africa from the north from about 500 AD onwards. It also confirmed the relative popularity of Indian women as wives among early colonists. It showed an unexpectedly frequent genetic contribution from the indigenous Khoekhoe and San populations and a greater West than East African genetic contribution in Afrikaners. Finally, there was a surprising lack of inbreeding.

Admixture during the formation of the Afrikaner population is recorded in genealogical sources. But these genealogies dont tell the full story, for several reasons.

Firstly, in the 17th and early 18th centuries some women used the toponym van de Kaap (meaning born at the Cape), irrespective of whether their parents were immigrants from Europe or slaves. Second, it has been suggested, but not recorded, that European farmers at the Cape had children with Khoe-San women.

Third, many of the children born in the Dutch East India Companys slave lodge had unknown European fathers. The slave lodge served as a brothel for passing sailors and other European men.

Several potentially important genetic source groups a substantial Muslim community, a small Chinese community and the local Khoe-San were not recorded because they were not Christian. And admixed couples would have been secretive about their relationships because marriages between slaves and Europeans were outlawed from 1685.

By comparing the Afrikaners in our study to 1,670 individuals from 32 populations across the world we found that 4.7% of Afrikaner DNA has a non-European origin. That may seem like a small percentage, but 98.7% of the Afrikaners were admixed.

Children whose parents are from different populations have one set of chromosomes from each population. With each generation the pairs of chromosomes one from each parent are snipped and pasted with one another; a process known as recombination. Repeated recombination results in shorter and shorter segments of DNA from the original populations.

By studying this effect, the age of the admixture was estimated to around 1681. Its around this time that colonisers began to settle at the Cape. In 1657, for instance, 142 employees of the Dutch East India Company were released from their employ to settle; 156 French Huguenots settled in 1688, and from 1675 yearly slave imports often exceeded 100 individuals. Therefore, this estimate aligns fairly well with genealogical and historical records of early colonial times at the Cape of Good Hope.

The admixture between European and Khoe-San was more common than church records suggest. In our study, though only 1.3% of Afrikaner genes came from the Khoe-San, most Afrikaners contained some Khoe-San genes.

The highest non-European contribution (1.7%) came from South Asia, or India. This reflects colonial mens stated preference for marrying freed Indian slaves during the founding years. A little less than 1% of Afrikaner genes have an East Asian (Chinese or Japanese) origin.

The contribution of West and East Africa is the lowest, at 0.8%. This is likely to stem from the almost 18,000 slaves imported from Africas west and east coasts. The fraction of genes from West Africa is slightly higher than from East Africa, reflecting the fact that while West African slaves were few, they arrived four generations before slaves from East Africa.

A common perception about Afrikaners is that they stem from very few ancestors, which would have resulted in inbreeding. Inbreeding results in long stretches of the paternal and maternal chromosomes being identical to each other. By looking at the lengths of identical stretches, it is clear that Afrikaners are as variable as the average European. This is in part due to admixture between non-Europeans and Europeans, but also because Europeans came from all over Europe.

The strongest European genetic contribution is from northwestern Europe, with the most similar population being the Swiss German population. This signal could also be interpreted as a mixture between German, Dutch and French populations as genealogical records indicate.

In conclusion, despite laws prohibiting mixed marriages from as early as 1658, and discrimination that culminated in the apartheid system, these genetic analyses confirm that most Afrikaners have admixed ancestry. Genealogical information has indicated as much, but these genetic findings are irrefutable.

See the rest here:
What genetic analysis reveals about the ancestry of South Africa's Afrikaners - The Conversation CA

People with rare diseases are being left behind by the Irish healthcare system, an Oireachtas committee has heard.

The joint committee on health met on Wednesday to discuss improving the lives of those affected by rare diseases and their families.

The committee heard that people with rare conditions struggle to access genetic testing and Irish patients have less access to new drugs compared to other European citizens.

Witnesses at the committee also said the health system needs to train more genetic consultants as a matter of urgency due to severe staff shortages.

A rare disease is a condition that affects less than one person in every 2,000.

According to Rare Diseases Ireland, there are roughly 300,000 people living with rare conditions in Ireland.

Vicky McGrath, CEO of Rare Diseases Ireland, said that diagnosis for people with a rare condition is often delayed for many years. Would we accept delayed diagnosis and treatment in other specialities? We all know what a delayed diagnosis for cancer patients means, yet it is accepted as normal for a rare diagnosis to take several years, she said.

Ms McGrath added 72 per cent of rare conditions are genetic in origin, but genetic testing, genetic consultation and genetic counselling is difficult to access in Ireland.

The Clinical Genetics service in Childrens Health Ireland (CHI) at Crumlin provides a diagnostic, counselling and clinical genetic testing service for children and adults affected by or at risk of a genetic condition. This service is the sole provider to the population of Ireland.

Ms McGrath said the HSEs Review of the Clinical Genetics Medical Workforce in 2019 revealed the extent of the issue.

There are currently just three genetic consultants in position in CHI at Crumlin. The HSEs 2019 Review indicates that there should be 15. The most visible knock-on effect is growing waiting lists.

As of March 2021, there were 3,999 people on the waiting lists for clinical (medical) genetics, up from 3,052 just one year earlier; 1,392 of these are children under the age of 16.

Typically, the priority waiting list is between 15 and 18 months and routine referrals wait over two years to be seen. As of March, there were 941 people on the waiting list for over 18 months, and 657 of these were under the age of 16, said Ms McGrath.

Access to drugs is another issue. There was a report published yesterday [on Tuesday] around access to medicines. Of the 47 orphan medicines that were approved by the European Medicines Agency between 2016-2019, eight of them were available in Ireland for reimbursement.

Ninety-six per cent of them are available in Germany, 85 per cent in Denmark, 72 per cent in England; Scotland, a similar country to our own, has 47 per cent of them available. Our system is hindering access... we are being left behind.

Dr Sally Ann Lynch, consultant clinical geneticist from CHI said that in Ireland, the training programme for genetic consultants was delayed for a decade.

I set up the training scheme in the Republic, but it was blocked by the Medical Practitioners Act for about seven years, we werent allowed to set up any new training schemes. Its also very difficult to recruit from abroad... Ive been trying to get a locum and will keep trying until the day I die.

The situation in Northern Ireland is far better than in the south, according to Dr Lynch. They currently have six geneticists, who were all trained in the North. They set up a training scheme, and really supported it.

However, Ms McGrath said because the North has more services, there could be an opportunity for cross-border co-operation, to reduce waiting lists.

Read the original here:
People with rare diseases being left behind by Irish health system - The Irish Times

Study shows link between mutations of GAS2 gene and ability to amplify incoming sound

Scientists have found a link between genetic mutations and hearing. Pictured: Cochlea in a mouse.

May 26, 2021

A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.

The U.S. National Science Foundation-funded scientists discovered that the protein encoded by GAS2 is crucial for maintaining the structural stiffness of support cells in the inner ear that normally help amplify incoming sound waves. Their findings, published in Developmental Cell, showed that inner ear support cells lacking functional GAS2 lose their amplifier abilities, causing severe hearing impairment in mice. The researchers also identified people who have both GAS2 mutations and severe hearing loss.

"Anatomists 150 years ago took pains to draw these support cells with the details of their unique internal structures, but it's only now, with this discovery about GAS2, that we understand the importance of those structures for normal hearing," said study senior author Douglas Epstein, a geneticist at Penn Medicine.

Two to three of every 1,000 children in the United States are born with hearing loss in one or both ears. About half these cases are genetic. Although hearing aids and cochlear implants often can help, these devices seldom restore hearing to normal.

One of the main focuses of the Epstein laboratory is the study of genes that control the development and function of the inner ear -- genes that are often implicated in congenital hearing loss. The inner ear contains a complex, snail-shaped structure, the cochlea, that amplifies the vibrations from sound waves, transduces them into nerve signals, and sends those signals toward the auditory cortex of the brain.

A few years ago, Epstein's team discovered that Gas2, the mouse version of human GAS2, is switched on in embryos by another gene known to be critical for inner ear development. To determine Gas2's role in that development, the team developed a line of mice in which the gene had been knocked out of the genome.

The prevalence of hearing loss in people due to GAS2 mutations remains to be determined, but Epstein noted that this type of congenital hearing loss is an attractive target for future gene therapy.

View original post here:
Discovery of a new genetic cause of hearing loss illuminates how inner ear works - National Science Foundation

GWAS and Polygenic Risk Scores (PGS)

Genome-Wide Associations Studies (GWAS) employ statistical means of describing the genome. They can be used to calculate polygenic risk scores or polygenic scores (they go by both names), which can tell you how your genetic constitution compares to others. It also can predict traits, including the risk of diseases caused by multiple genetic combinations. (Heres more on GWAS and PGS).

But while your PGS can tell you that you may be at a higherriskof, say, coronary heart disease it wont tell youwhenyou might get sick or evenifyou will get sick at all. The most your PGS can tell you is yoursusceptibilityto disease. Nor does PGS factor in contributory causes like environmental insults or lifestyle, diet, or stress, which also influence disease onset.

Choice over Chance

PGS can tell you whats bad about your genome but it can also tell you whats good about it. For reproductive entrepreneurs, this translated into using these scores to select the best embryo for implantation following In Vitro Fertilization (IVF). At least one Americancompanyadvertises the technology to choose the healthiest embryo amongst the litter of recovered fertilized eggs.

It doesnt take much imagination to conjure the creation of a PGS for intelligence(some reports say it already exists and is available for the wealthy[1, 2])or aesthetics, using an algorithm for height, body-mass index, eye and hair color, skin tone, facial symmetry and Fibonacciproportionalityof features, or athleticism, including genetic markers for endurance, muscle mass, and strength. These scores would allow prospective parents to choose the embryo genetically destined to be the best looking, smartest,healthiest, or most athletic of their offspring that is, if you dont place much importance on environmental and personality factors, such as drive, discipline, resilience, and motivation. (Although at least one evolutionary geneticistclaimsthat even these factors are also genetically influenced [3])

Legally, in the United States, there is no problem using PGS to select the best embryo. Medically, it entails no additional risk to the embryo - IVF embryos are routinely screened for genetic markers that compromise gestation, anyway. So, the question remains: should this be done?

Bioethics and Beneficence

At least two noted bioethicist-scholars advocate in favor of genetic selectivity of embryos- based on an idiosyncratic reading of beneficence (the obligation of an individual to act for the benefit of another), one of the four bioethical principles offered by Beauchamp andChildress.

Julien Savulescuclaims it is a moralobligationfor prospective parents to choose the best child, meaning the most advantaged child, or at least the one with the greatest chance of having the best life, under the theory of procreative beneficence. Considerations of the future implications of such use amply depicted in fiction scenarios are ignored.For Savulescu, the concept ofwhochooses what constitutes best is unimportant. As to whether parents may be swayed by fashion, superstition, and outrageous conception of the good life, he (wrongly) claims there are legal constraints that aim to prevent the most egregious parenting choices.

Professor John Robertson holds a similar opinion invoking procreative liberty, which allows using an IVF procedure even if it increased the childs risks of injury. To Robertson, children born with these afflictions would not be harmed because the alternative future for them would be non-existence, [2] a belief that I do not share and havewrittenat length.

The Rights of the Child (Autonomy)

Autonomy, another ethical principle proffered by Beauchamp and Childress, is the right of self-determination.Those disagreeing with using PGS to select the best embryo claim the child has a right to an open future, and a parent who chooses the embryo scoring highest on one matrix might be directing the child in a direction adverse to what the child might have chosen herself.

Indeed, while parents typically chose a partner that facilitates a reproductive likelihood in a particular direction good parents dont push their offspring down a particular path (lest they spend years and big bucks on a shrinks couch undoing this primordial programming). To allow parents to choose their childs precise genetic destiny from the moment of conception trespasses on the childs right to choose what life she or he would like.

Social Justice to treat everyone equally and equitably

The third Beauchamp and Childress principle is justice, encompassing social justice. Here, the potential for societal danger conjured by the technology seems to have been ignored entirely by proponents of using PGS for embryo selection. Until these technologies can be made available to everyone, they will be the province of the rich whose children often begin life healthier by virtue of better environments, which is also said to boost intelligence scores (NB this isnotto be confused with intelligence).With plastic surgery, they are prettier. With drugs, their athletic performance is enhanced. The disparities of health outcomes from socio-economic determinants are well-studied, and the availability of this technology to the rich, when not available to all will only further expand the divide.

But even if the technology were available to all lets say to enhance intelligence, it wouldnt make one child any smarter compared to the next if she werent already destined to be.

If everyone might be genetically enhanced allwho are now smarterwould still be smarter genetically,their environments would still differ leading to the same state of affairs at least relatively speaking [4]

Non-Maleficence IVF can be dangerous

The final Beauchamp and Children ethical principle is non-maleficence do no harm. One might question using PGS at all, as it requires submitting to IVF. While IVF is a godsend to address infertility (and perhaps to select for children with certain immunological profiles to enable stem cell transplantation for sick siblings, as Ive previouslywritten), some suggest that IVF should not be routinely countenanced where infertility is not an issue as the procedure entails rare risks of its own both to mother and child-to-be, being responsible for a slight increase in birth defects among other problems(4).

Truth in Advertising and Biological Validity

Most of those in the know recognize that PGS are predictive only for populations.

We can certainly use genetics to look at statistical effects across populations, but this will give at best very fuzzy predictors for individuals.

Dr. Kevin Mitchell, geneticist [1]

Perhaps when there is only one prize being contested for, say, health, it might make sense to allow parents to choose the embryo with the probability of being healthiest (defined according to todays technology). But when we include the choice between various packages all involving probability functions no definite outcome can be predicted. How could one reasonably choose between an embryo with a 90% chance of being healthy or one with a 60% chance of being more intelligent than her siblings?

Perhaps more egregious is the failure to recognize the impact of pleiotropism, meaning that one gene has multiple effects. This consideration is important both in CRISPR gene-editing and PGS determinations.

Pleiotropisms come in two varieties, vertical and horizontal. In the first, the genetic variant under question affects one trait, say cholesterol, which in turn affects others, like the risk of heart disease. Of more concern are the horizontal variants, where one gene has multiple non-related effects. So, say you want to create a child with the least risk of mental health issues including a minimal risk of schizophrenia. Genes associated with reducedschizophreniarisk are also associated with both low and high body mass meaning if you choose against schizophrenia, you might also be selecting fora child likely to be obese. Since we arent conversant yet with the extent of genetic pleiotropisms, the unanticipated consequences of using PGS strongly cautions against its use at present.

Morality and Humanity

The magic promised by these technologies seems to have fairy-dusted the eyes of even the most intelligent.This raises the phantasm of PGS or gene-editing to cure or eliminate diseases, like schizophrenia, Lou Gehrigs disease, dyslexia, or dwarfism. How wonderful, we think, to eliminate these diseases from the face of the Earth. Perhaps not.

Had we given the parents of embryos containing markers for these diseases the chance to avoid birthing children with them, society would have been deprived of the contributions of John Nash (the Nobel prize winner in Mathematics), theoretical physicist Stephen Hawking, Carol Greider, the Nobel Laureate who discovered telomerase, and Professor Charles Steinmetz, the electrical engineering genius who boosted our capacities in electrical power systems, just to name a few who suffered from these conditions. And people who dont achieve high scores on any PGS rubric, like my friends dear daughter, would be denied existence if these scores were in common use - prevented from enriching and brightening our lives with their smiles, kindness, and their good cheer.

[1] Hannah Crichtlow,The Science of Fate,Hodder Press

[2] O. Carter Snead,What It Means to be Human, Harvard University Press

[3] Robert Plomin, Blue PrintHow DNA Makes Us Who We AreMIT Press (2018

[4]Genetically-Engineered Begots, Have-Nots, and Tinkered Tots: (High Scoring PolyGenic Kids as a Heredity-Camelot) - An Introduction to the Legalities and Bio-Ethicsof Advanced IVF and Genetic EditingSSRN.com3851431, Chicago-Kent Law Review (forthcoming) 2021

More:
'Genelection': Should We Select Children Based on Their Genetic Scores? - American Council on Science and Health

A blind man who could only perceive the faintest bit of light can now perceive fuzzy shapes, thanks to gene therapy and a pair of specially engineered goggles.

The man was diagnosed with a condition called retinitis pigmentosa 40 years ago, at the age of 18, according to a new report, published Monday (May 24) in the journal Nature Medicine. People with retinitis pigmentosa carry faulty genes that, due to many mutations, cause the light-sensitive cells in the retina at the back of the eye to break down, according to the National Eye Institute (NEI).

These genes would usually code for functional proteins in the retina, but instead fail to build those proteins, or make abnormal proteins that malfunction or produce substances that directly damage the retinal tissue. The condition affects roughly 1 in 4,000 people worldwide, according to the NEI, and can sometimes lead to complete blindness, as occurred in the 58-year-old patient in the new study, BBC News reported.

Related: 12 amazing images in medicine

In an attempt to treat the man's vision loss, scientists inserted genes that code for a light-sensing protein into a modified virus, then injected those genetically tweaked viral vectors into one of his eyes, the researchers reported. The protein, called ChrimsonR, is a engineered version of a light-sensitive protein found in unicellular algae, which allows the single-celled organism to detect and move toward sunlight, MIT Technology Review reported.

ChrimsonR belongs to a family of light-sensitive proteins called channelrhodopsins, hence the added "H" in crimson, and has been modified to react to colors within the reddish end of the color spectrum, namely amber light. By injecting genes for ChrimsonR into the retina specifically into retinal ganglion cells, a kind of nerve cell that sends visual signals to the brain the team hoped to make these cells sensitive to yellow-orange light, MIT Technology Review reported.

Here's where the special goggles came in. The goggles pick up changes in light intensity from the environment and then translate that signal into an intense, amber image that gets projected straight onto the patient's retina, with the aim of activating ChrimsonR. Months passed before a significant quantity of ChrimsonR accumulated in the man's eye and began to alter his vision, but eventually, he began to perceive patterns of light with help from the goggles, BBC News reported.

"The patient perceived, located, counted and touched" different objects using his treated eye, alone, and while wearing the goggles, the researchers wrote in the study. For instance, the patient could perceive a notebook and cups placed on a table in front of him, although when asked to count the cups he did not always give the correct number, according to MIT Technology Review.

Prior to receiving the therapy, the man could not detect any objects, with or without the goggles on, and following the injection, he could only see while wearing the goggles, since they convert all light into an amber hue, the researchers reported.

In addition to the notebook and cups, the patient reported being able to see the painted white lines at a pedestrian crossing, the BBC reported. "This patient initially was a bit frustrated because it took a long time between the injection and the time he started to see something," first author Dr. Jos-Alain Sahel, an ophthalmologist and scientist at the University of Pittsburgh and Institute of Vision in Paris, told the BBC. The patient began training with the goggles about 4.5 months after his injection and only started reporting improvements in his vision about 7 months after that, the team reported.

"But when he started to report spontaneously he was able to see the white stripes to come across the street you can imagine he was very excited. We were all excited," Sahel told the BBC.

Even now, the man's vision still remains fairly limited, in that he can only see monochromatic images and at a fairly low resolution. But "the findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible," senior author Dr. Botond Roska, founding director of the Institute of Molecular and Clinical Ophthalmology Basel at the University of Basel, told BBC News. ("Optogenetics" broadly describes the technique of using light and genetic modification to control the activity of neurons.)

Of course, although these initial results are exciting, the study is limited in that only one patient has received the treatment so far, James Bainbridge, a professor of retinal studies at the University College London who was not involved in the study, told the BBC.

Read more about the research in BBC News and MIT Technology Review.

Originally published on Live Science.

Excerpt from:
Genes from algae helped a blind man recover some of his vision - Livescience.com

A blind man who received a gene from algae in one eye reaches for a notebook with the help of special goggles.

By Jocelyn KaiserMay. 24, 2021 , 12:20 PM

A blind man who received a gene for a light-sensing algal protein can now see and touch objects with the help of special goggles, researchers report today.

His vision gains are modesthe cannot see colors or discern faces or letters. But if the treatment helps other study participants, it may offer advantages over other vision technologies for severely blind people. And for neuroscientists, the result is a milestone: the first published report of using a relatively new technology called optogenetics to treat a disease in people.

Its not the kind of vision people dream of, but its a big step, says Jean Bennett of the University of Pennsylvania, who works on gene therapy for blindness but was not involved in the study.

Optogenetics uses light to control neurons. Scientists add the gene for a light-sensitive protein called an opsin from algae or bacteria and then shine a light on the cell to trigger the opsin to change shape, which switches the neurons activity on or off. Since it was developed nearly 20 years ago, optogenetics has mostly been used as a tool to study brain circuitry in animals. But researchers hope it can one day treat diseases such as Parkinsons and blindness.

The eye is the simplest place to start because it is small and easy to access, study co-leader Botond Roska, a physician-scientist at the University of Basel, said at a press conference last week.

The patients in the studya clinical trialhave an inherited disease called retinitis pigmentosa and have lost the retinal photoreceptor cells that use human opsins to turn light into electrical signals relayed to the brain. But their eyes still have the ganglion cells that route these signals to the brain via the optic nerve. That means the patients could potentially gain vision by giving these cells a microbial opsin.

The first volunteer was a 58-year-old French man who began going blind 40 years ago. When the experiment started, he could sense light but could not distinguish shapes. He received an injection in his worse eye of a harmless virus called an adeno-associated virus, which carried the gene for an opsin from algae. The researchers waited a few months for the ganglion cells in the patients eye to begin to produce the new protein. Then they began to train him to use special goggles that amplify incoming light from an image and focus it on the retina in the amber wavelength sensed by the opsin.

Within a few months, the man reported he could see the white stripes at a pedestrian crossing while walking outside wearing the goggles. He was very excited, although perhaps not as much as we were, study co-leader Jos-Alain Sahel, a physician-scientist at the University of Pittsburgh School of Medicine and the Vision Institute in Paris, said at the press conference.

Then the man started to pass lab tests: He could usually find and touch dark objects set on a white table, such as a notebook or box of staples, that he could not see without the goggles. And he could count up to three glass tumblers (see videos below). When the patient wore an electrode-studded cap that measures brain activity, the signals showed activity in the visual cortex, the part of the brain involved in seeing, the team reports today in Nature Medicine.

The man also told the researchers his daily life has improved. Wearing the goggles, he said, he can more easily find a plate or phone or detect furniture or a door.

The seven other patients treated so far in the trial havent been able to complete training with the goggles because of the coronavirus pandemic. Some are receiving higher doses of the viral vector that could help them see in more detail, as could tweaks to the goggles, the researchers saythough none of these improvements would allow for color vision.

Its fabulous that they got this to work in humans, says neuroscientist Pieter Roelfsema of the Netherlands Institute for Neuroscience. Right now, the only approved treatment for such patients is a device that sends signals from a camera mounted on a pair of glasses to electrodes implanted in the eye. It can improve light perception and allow some people to see shapes, but it requires surgery. Roelfsemas lab is developing a brain implant that has helped monkeys see letters, but it would be much more invasive than an eye injection, he notes.

The company that sponsored the trial, GenSight Biologics, isnt the only one working on optogenetics for blindness. RetroSense Therapeutics launched a trial 5 years ago but hasnt reported results. Another company, Bionic Sight, reported in March in a press release that four patients can now detect light and motion when looking into a device similar to a virtual reality set.

Read the original here:
Blind man regains some vision, with help from light-sensing algal protein - Science Magazine

(BPT) - As one of the worlds top paratriathletes, Amy Dixon is always looking forward. Even though an autoimmune disorder has taken away most of her ability to see, she has extraordinary vision for reaching her goals.

This summer, shell head to Tokyo. For Amy, its an incredible journey that would have seemed unimaginable two decades earlier.

Vision troubles come to light

Amy first noticed her vision problems as a 22-year-old college student. She struggled to see clearly in a darkened room. Driving at night, she failed to notice oncoming cars. The signs were clear that something was wrong with her eye health, but Amy wasnt convinced.

I had 20/20 vision as a kid, so I dismissed the problems I was having as being related to some other condition like migraine, says Amy, a migraine sufferer since she was a teen. I was ignoring what was obvious that something was wrong with my eyes.

After scheduling an appointment with an ophthalmologist, Amy learned she had uveitis, which is a form of inflammation inside the eye. Amys doctor told her that the disease had already put her eyesight in serious jeopardy. He said that 70% of her peripheral vision had been lost and she would need to begin treatment immediately or risk going blind within 10 years.

Unfortunately, I waited too long before having my eyes examined and uveitis had already attacked my vision. When the diagnosis sunk in, I thought I was destined to going blind, adds Amy.

Becoming her own advocate

Rallying behind a forward-looking attitude that would ultimately become her calling card, Amy confronted her condition head-on. Working with her doctor, she began an aggressive treatment regimen. While uveitis would eventually take 98% of her vision, the treatments succeeded in slowing down progression of the disease.

A new diagnosis and the athlete reemerges

With her uveitis in remission, Amy received a second vision diagnosis: she now had developed glaucoma as a result of her treatment. Resilient and determined to keep her life moving forward, Amy began treating her glaucoma.

Along the way, she reengaged in sports and took up swimming, a favorite activity for the former competitive high school swimmer. When a friend introduced her to triathlons (swimming, running and biking), Amy was hooked. She completed her first triathlon in 2013 and today, she is the reigning ITU Aquathlon World Champion and a seven-time ITU Triathlon Gold Medalist. When the competition in Tokyo starts in August, Amy will race toward the finish line the same way she approaches life: by overcoming the setbacks in her path. Its an important lesson she is eager to share.

There is always a way forward, says Amy. I encourage people to maximize the strengths they have and find creative ways to do the things they want. It may not be the way you wish for, but if you are open to learning, you can do great things.

Amys prescription for better eye health

Amy views her journey as a cautionary tale and she encourages everyone to be proactive in taking care of their eyes.

Uveitis progressed quickly in impacting my vision because I waited too long to see a doctor and wasnt diligent about getting my eyes examined annually, says Amy. Pay attention to your eyes. If you suspect you have a vision problem, then see an eye doctor right away.

As she continues to manage her glaucoma, Amy also urges people, particularly young adults, to be wary of a disease that can sneak up without symptoms and is the leading cause of irreversible blindness.

A dilated eye exam could save your sight, says Amy. The power is in your hands, so be your own advocate for achieving better eye health.

If youve been diagnosed with glaucoma or are caring for someone with glaucoma, a great resource is Understanding and Living with Glaucoma. This free booklet is published by the Glaucoma Research Foundation, with support from Aerie Pharmaceuticals. It can be downloaded or ordered (in English and Spanish) at http://www.glaucoma.org/booklet.

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Top paratriathlete carries the torch for healthy vision to Tokyo - liherald

The vision of a man who lost his sight because of retinitis pigmentosa (RP) has been partially restored with optogenetic therapy in the first reported case of functional recovery in a neurodegenerative disease following this novel treatment.

"It was breathtaking to witness the first recovery of some visual function in a blind patient," Botond Roska, MD, PhD, said in a news release.

"We have worked on optogenetic therapy in the lab for 16 years, and now seeing the proof of concept in a patient is a unique experience," added Roska, founding director of the Institute of Molecular and Clinical Ophthalmology, Basel, Switzerland, and co-founder of GenSight Biologics, the company developing the therapy.

The case was reported onlineMay 24 in Nature Medicine.

The 58-year-old man's blindness was caused by retinitis pigmentosa (RP), a neurodegenerative eye disease in which loss of photoreceptors can lead to complete blindness.

As part of the phase 1/2a PIONEER study, the patient received an intraocular injection of an adeno-associated viral vector encoding the optogenetic sensor ChrimsonR into one blind eye. The team also engineered light stimulation goggles outfitted with a camera that captures and projects visual images onto the retina.

Training with the goggles began nearly 5 months after the injection, thereby giving ChrimsonR expression time to stabilize in ganglion cells. Seven months later, the patient began to report signs of visual improvement.

"In this type of study, we are really learning from the patients themselves. They are really like experimentalists telling us what they are seeing with restored vision," lead investigator Jos-Alain Sahel, MD, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, said during a press briefing.

The patient was able to perceive, locate, count, and touch different objects using the vector-treated eye alone while wearing the goggles, the team reports. The patient could not visually detect any objects before injection with or without the goggles or after injection without the goggles.

The patient also reported significant improvements in his ability to conduct day-to-day activities, such as navigating in outdoor and indoor environments.

During visual perception, multichannel electroencephalographic recordings showed object-related activity above the visual cortex.

"So it was possible to show in this patient that visual behavior was correlated with brain activation corresponding to visual function," Rosko said.

A video of the patient performing the tests, which was submitted as supplementary material to Nature Medicine, can be viewed at http://www.gensight-biologics.com.

"This is a significant milestone, and undoubtedly further refinements will make optogenetic therapy a viable option for many patients in future," Robert MacLaren, MBChB, DPhil, professor of ophthalmology, University of Oxford, Oxford, United Kingdom, said in a statement from the nonprofit UK Science Media Center.

Also weighing in, James Bainbridge, MBChB, PhD, professor of retinal studies, University College London, London, United Kindgom, said, "This exciting new technology might help people whose eyesight is very severely impaired. It is a high-quality study. It is carefully conducted and controlled.

"The findings are based on laboratory tests in just one individual. Further work will be needed to find out if the technology can be expected to provide useful vision," Bainbridge noted.

The study was funded by GenSight. Several authors have disclosed financial relationships with the company. MacLaren is a scientific advisor to NICE on retinal gene therapy. Bainbridge has disclosed no relevant financial relationships.

Nat Med. Published online May 24, 2021. Full text

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A First: Blind Man's Sight Restored With Optogenetic Therapy - Medscape

Optogenics was used to partially restore a blind mans eyesight; the FDA could soon approve Modernas COVID-19 vaccine for use among adolescents; HHS calls for a new investigation into COVID-19s origins.

Light-catching proteins were grown in the eye of a blind man in France who, with the help of special goggles, can now see blurred outlines of objects, reports an article in Nature Medicine. This novel form of gene therapy is optogenics, and these results mark its first successful use against a neurodegenerative eye disease, which rob[s] the eyes of essential proteins required for vision. The treatment transforms ganglion cells into new photoreceptor cells, with the scientists also utilizing algae-derived proteins to sensitize nerve cells to light.

Hoping to gain approval for its COVID-19 vaccine among adolescents aged 12 to 17 years, Moderna will submit effectiveness data to the FDA in June, reports The New York Times. Currently only available for persons 18 years and older, the new vaccine data comprise results from 3732 participants. Most notably, efficacy was shown to be 100%, following no reports of symptomatic COVID-19 in the two-thirds who received both doses. Pfizers vaccine was similarly approved for use among adolescents aged 12 to 15 years on May 10.

Following US intelligence reports that Chinese virology experts were seriously ill before cases of COVID-19 were ever reported in December 2019, HHS Secretary Xavier Becerra is seeking a more transparent phase 2 investigation into the beginnings of the COVID-19 pandemic, according to Reuters. This new effort should again be headed by an international team, he added. Speaking for the World Health Organization (WHO), whose previous report on the pandemics origins was met with much criticism, spokesperson Tarik Jasarevic noted that the next step will be to present a proposal to WHO Director-General Tedros Adhanom Ghebreyesus on studies that cover early detection of cases and clusters and food chain transmission, among others.

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What We're Reading: Restoring Eyesight; New Moderna Vaccine Indication Possible; COVID-19 Origin Investigation - AJMC.com Managed Markets Network

In late 2020, scientists at Trinity College Dublin found that gene therapy could successfully protect he visual function of mice who were treated with a chemical targeting the mitochondria and were consequently living with dysfunctional mitochondria. The scientists also found that their gene therapy improved mitochondrial performance in human cells that contained mutations in the OPA1 gene meaning that this could also work in humans, one day.

Professor Farrar, part of the OPA1 study, said at the time: It is important to highlight that there is still a long journey to complete from a research and development perspective before this therapeutic approach may one day be available as a treatment.

Yesterday (24 May), a journal article was published that documented the restoration of partial vision to a human being with blindness.

Retinitis pigmentosa (RP) is a progressive, inherited, monogenic or rarely digenicblinding disease caused by mutations in more than 71 different genes. It affects more than 2 million people worldwide. With the exception of a gene replacement therapy for one form of early-onset RP caused by mutation in the geneRPE65.

Currently,there is no approved therapy for RP.

The main subject of this study was a 58-year-old man who was diagnosed with Retinitis pigmentosa, 40 years ago. When he begun the experiment, he could sense light only if it was there or not.

The study is the first evidence that the injection of optogenetic sensor-expressing gene therapy vector (gene therapy) in combination with light-stimulating goggles, specially engineered to provoke the eye to see, can create a partial vision.

It took seven months after the beginning of visual training for the man to be able to perceive objects. The researchers say that this training meant teaching the patient to become aware of the direction of his gaze and to control his eye movements to be able to look straight into the light beam projected by the goggles.

The goggles projected light straight into the eye. But before this stage of light and glasses, an injection was given.

A specific type of virus can provoke the brain to build light-sensitive channels. The team gave this man a protein that is sensitive to amber light, using viruses to deliver the protein directly into the retina. This process is vaguely, loosely similar to how some vaccines work the good stuff is delivered via a virus, which then provokes the immune system to create a really useful response.

Botond Roska, an ophthalmologist at the University of Basel and a co-author of the new study, said: The brain has to learn a new language.

The team placed objects on a white table in front of him, and asked him to identify where they were. After visual improvement, it was still the larger objects that were more easy for him to locate.

The team will now begin building lighter, less cumbersome goggles and trying this process out on others to see how the results hold up. A freely available therapy for blindness is still distant, but it more partially visible than it ever was.

Read the full study here.

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Scientists successfully use gene therapy to restore eyesight restore eyesight, gene therapy - Open Access Government

Glaucoma. Cataracts. Blurry vision. Age-related Macular Degeneration (AMD). Myopia. If not detected and treated early, these are vision problems in the elderly that could lead to partial or complete vision loss.

I understand how it feels because I was once in your shoes. My name is Mrs. Damilola, and for several years, I used to battle eye problems that made me had blurry, unclear and poor vision.

After trying several eye drops and glasses for over 3 years, I noticed that instead of improving, my eye condition had worsened.

After an eye exam, I was diagnosed with glaucoma in the right eye.

You see, when patients are diagnosed with glaucoma, one of the most common treatment options is a prescription eye drop medication that lowers eye pressure, which prevents damage to the optic nerve and helps to preserve vision.

Several types of drugs lower pressure.

The choice depends on a lot of factors, including the type of glaucoma, its severity, the pressure goals the doctor wants to reach, and the patients other medical problems.

For my own case, the doctors recommended eye drops, drugs and even eye surgery.

Eye surgery which was the best long term treatment was really expensive, and even comes with its own side effects after several months or years.

So, I decided to start using eye drops just so I could manage to see because at this point, I was literally going blind.

You see, the problem with eye drops is that most of them came with serious side effects, so one has to continue changing them until you see the most suitable one.

Sadly, within few months I used about 4 eye drops.

While for over the counter eye drugs, they take almost forever to work and give you perfect eye so you keep on spending and spending on the drugs for several years.

So, I decided to start searching for an alternative solution, something herbal that comes with no side effects and will naturally reverse glaucoma, cataracts, near-sightedness, and other severe eye problems.

But the entire situation changed when I came in contact with an old time friend, Uwem, he used to work in a particular eye clinic in Abuja .

We have been friends since our NYSC days for over 7years till date. Although, it has been a long time we spoke because he relocated to Canada to continue his profession.

We started talking as usual, and he asked me about my eye condition, if it has improved for better.

I explained how I was already tired of looking for solution to the eye problem, and how I have spent lots of money on drugs, supplements, eye drops and glasses, desperately looking for solution but without any improvement, insteadI was almost getting blind instead!

He explained that most people wearing eye glasses suffer from myopia (nearsightedness), glaucoma and cataract.

He later explained that there ispermanent cure for most of the eye problems, and natural remedy that helps treat and reverse glaucoma, cataract, myopia and other severe eye problems.

Most of these cases he has handled in the eye specialist clinic he works with in canada, and his patients improved their eyesight, and they never had to wear glasses or go for expensive eye surgery again.

But sadly, the solution is very rare to find because its imported, specially for Eye clinics in the USA, UK, Canada and some developing African countries as well.

That was when he introduced me to aspecial herbal teathat have been in existence for several years, that help cures severe eye problems, reverse cataract, glaucoma and myopia.

At first, I was shocked and surprised. But he told me the exact plant extracts contained inside the herbal tea, and how they improves overall eyesight health and restores vision.

I quickly pleaded with him to help me send a complete supply of the herbal tea. The next morning, I quickly sent the money to him.

After about a week, the complete supply of the herbal tea was delivered to me.

Immediately, I started it using it as prescribed (serving size of 1 tea bag in morning, and 1 tea bag in the evening).

To my surprise, in less than 2 weeks of using it, it was almost like a miracle cure to my eyesight problem and it helped improve my vision.

My eyes became crystal clear, my vision was never blurry again, and I started to feel that my glaucoma was gone.

To ensure it was really effective,I had to give the same herbal tea to about 2 of my colleagues in a different branch office, and some of my relatives using spectacles just to confirm if it could help them improve their eye health and cure eye problems.

Within 30 days or less of using it, all of them came back to testify that this herbal tea helped them improve their eyesight and cure eye problems like cataracts, glaucoma, near-sightedness, hyperopia, and blurry vision, and they didnt have to continue wearing glasses anymore.

To ensure that this solution really works, I went to my eye doctor, just to be completely sure be about it.

After several eye exams, he confirmed that my eye was much better than before, and that the herbal tea also contained some ingredients that are very rare to find, but are highly effective in improving eyesight naturally.

Without wasting time, let me introduce you to the Herbal Remedy that Has Helped me and over 642 Nigerian men and women Improve Eyesight, vision and PROVEN to cure Severe Eye Problems

Eye Restore Tea

Eye Restore Teais a herbal remedy formulated to treat and permanently cure all kinds of eye problems such as glaucoma, myopia, hyperopia, cataracts and lot more

It helps restore your eyesight, promotes eye health and improves vision.

Eye Restore Tea is made up of finely blended plant herbal extractssuch as bilberry extract, chrysanthemum, chinese wolfberry, cassia seed and green tea.

A pack of eye restore tea contains 20 tea bags, which you use daily, morning and evening.

The best part is that the Eye Restore tea is purely herbal, comes without any side effect and curative for the treatment of most eye problems. And within few days of taking this herbal tea, you can start seeing improvement in your vision and eye health.

Eye Restore Tea has the highest standard of approval by International FDA bodies. Its produced in the best, biggest and professional tea manufacturing factory withcertificate of GMP Good Manufacturing Practice, HACCP, Food Production License, QS etc.

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Do you want to dump your glasses, and also put an end to using expensive eye drugs that takes almost forever to work?

Then pay attention to this.

Eye Restore Teais made of herbal extracts, that after several years of research, scientist prove that these herbs help eliminate any form of eye problems, gives a crystal clear eye, improves eye sight and vision naturally.

Heres how this special herbal tea works

When you order for the Eye Restore Tea, each pack contains 20 tea bags. Put 1 tea bag in the cup, add boiled water, you can add honey or lemon (depending on your taste). Drink the tea in the morning when you wake up, and in the night before bed.

Within 3 weeks or less, I can guarantee that it will cure whatever eyesight problem you are experiecing at the moment. It will improve and perfect your eyesight, you will be able to read better anytime of the day. It will help you see comfortably without your glasses.

THATS how powerful it is.

Right now, my eyes are crystal clear, no more blurry vision, I see clearly without my eye glasses, glaucoma has gone, and no more cataracts.

And after this worked for me, Ive gone to share this amazing eye restore secret with almost everyone (family, friends and collegues) that I know that uses spectacles, or have severe eye problems for many years.

And once they started using Eye Restore Tea, they experienced similar results, their joy knew no bounds and they were able to dump their glasses, save money on expensive drugs and eye drops.

While it has also helped them to have a much improved vision, eyesight and totally natural cure to glaucoma, cataracts, myopia, hyperopia, eyestrain and other eye problems.

And to Prove It, Here Are What People Who Have Used theEye Restore Tea Are Saying About it, and How TheyveSeen Great Results Using it

For over 3 years of having glaucoma in my right eye, Ive been eye drops that my eye doctors prescribed. After using it for a long time, I noticed that the eye was becoming useless, and I only had my left eye for all purpose.

Until I came accross Eye restore tea, I decided to use it instead. Within few weeks, I began to see better. I went to the Eye center, and after the examination doctor say that my eye has improved and the IOP has dropped.Thanks so much for this wonderful solution that saved my eyes

James Gwarimpa, Abuja=================

I suffered from Glaucoma for about 2 years now, and I had to use eye drops to control it. But the eye drops have serious side effects that made me uncomfortable so I had to keep changing them and used 5 types of eye drop.

Luckily, a friend introduced me to Eye restore tea, so I decided to give it a trial. My vision now have improved up to the extent that I no longer need spectacles or any eye drop again.

Mrs. Tolulope Ketu, Lagos===============

Immediately after my cataract sugery, I used to have a foggy or cloudy vision and colors are not vivid as they were. I got corrective sight vision lens, and after many months I still get this blurry vision sometimes which is frustrating.

My wife helped me order for the Eye Restore tea, and I decided to use it, within 6weeks, my eyesight is clear and has corrected itself with time. Ive gone on to recommend this for my elder brother with similar eye problems. Thanks so much for this miracle tea

Mr. Victor Onitsha, Anambra

============

After seeing an opthalmologist for an eye exam, it was revealed an eye pressure of 25 in both eyes, and my right eyes looked suspicious for glaucoma. I continued using eye drops.

I stumbled upon your eye restore tea product, and decided to go for it. Within few weeks of using the tea, am seeing obvious changes, and finally stopped using eye drops. i will keep you updated on my progress, really appreciate this!

Olawale warri, Delta

Truth is, I could load more and more testimonials from these people who have used the Eye Restore tea but it will only be saying the same thing this product works like crazy!

As you have seen, these people were once like you suffering form similar eye conditions that were a threat to their vision, but they took action got the Eye Restore tea and cured severe eye problems.

As we speak lots of people are raving about this natural miracle eye treatment solution. I wonder what happens when people begin to post experiences and testimonials on social media (Facebook, Instagram, Twitter etc.)

Customers will continue telling friends, and the news keeps on spreading like wildfire.

By word of mouth, theres so much huge demand, supplies are going off the shelf fast even my colleagues and inner circle friends picked up 143 packs of the Eye Restore Herbal tea.

So, there are just 257 packs remaining.

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We have no idea when the next stocks will arrive, so get your supplies now while you can.

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I cannot assure you will get it at the same discount price if you procrastinate.

SO WHATS IT COST?

Let me ask you whats something like this worth?

What would it be worth to be able to see clearly again, without spending lots of money on presciption pills, eye drops or expensive surgery?

What would it be worth to improve all aspects of your vision using a natural solution that completely reverses glaucoma, cataract, shortsightedness and other eye problems?

A natural solution that makes you never scared again about having dry itchy eyes, blurred vision, or even worse going blind.

So that, you will be able to enjoy your eyes, watch tv, read newspapers, reports, documents without depending on your glasses ever again.

Eye restore tea, is like giving you a brand new set of perfect eyeballs faster, more safer, and more easily.

What would that be worth?

You see, you wont pay a lot for this miracle herbal tea.

And if you go ahead and get it now, you will enjoy

=> the discount introductory price

=> 100% FREE Shipping to every state in Nigeria

=> Payment at the point of delivery (or at your DOORSTEP)

This means you will pay for the products only when it has been brought down to you face to face by our courier company or delivery man.

Heres a breakdown of the price (depending on the supply you are ordering for)

Eye Restore Tea 1 Month Supply3 packs (60 tea bags)

]

Discount Price = N24,000

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62year old Lagos woman Reveals Eye treatment Remedy that Reverses Glaucoma, Cataracts and Improves Vision without surgery or eyedrops - Vanguard

One of the studys researchers demonstrating the set-up used to confirm the success of the gene therapy treatment, including the use of specialized goggles seen above. Image: Sahel, et al., Nature Medicine

Researchers say their experimental gene therapy was able to partially restore a mans eyesight 40 years after he lost most of his vision. The findings are exciting, but the authors of the new study caution that more research will be needed to confirm the effectiveness of this treatment.

Gene therapy has emerged as a promising approach for largely incurable ailments that are often caused by defective genetic mutations. It can include editing the genes of cells collected from the body in the lab, then putting them back in (CAR T-cell therapy for cancer is one example of this) or editing cells inside the body directly, also known as in vivo gene therapy. In 2017, the very first in vivo gene therapy was approved by the Food and Drug Administration. The treatment, called Luxterna, helps prevent people with inherited retinal dystrophy, a rare genetic disorder, from losing their vision completely.

Luxterna is only helpful for people with a form of retinal dystrophy caused by a specific mutation, though, and only before they completely lose their functional retinal cells, which usually happens before adulthood. So scientists have been trying to develop other kinds of gene therapy that can broadly help people with these retinal disorders, no matter the mutation causing it and long after its onset. One approach being tested now relies on something called optogenetics. The idea is to edit nerve cells collected from a patient so that they respond to light in a particular way, which should then essentially turn these cells into a version of the photosensitive cells normally found in the retina that allow us to see.

Researchers in Europe and the U.S. have been working on one of these therapies as part of a Phase 1/2 clinical trial called PIONEER. Like other gene therapies, this treatment uses a neutered adenovirus to deliver its payload to cells: genetic code that allows cells in the retina to produce a protein called ChrimsonR that responds to amber light. They then use specialized goggles to collect information from the outside world and turn that information into pulses of amber light. The hope was that these pulses would then allow the gene-edited retina, and by extension the brain, to perceive visual information the way people with undamaged retinas canthus restoring sight.

In a paper published Monday in Nature Medicine, the team detailed the case of a 58-year-old patient involved in the trial, who regained eyesight following treatment, albeit with some caveats.

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The patient, who did retain an ability to perceive light even with his disorder, was given the treatment in one eye. In that eye, and while wearing the goggles, he became able to see nearby objects, to the point where he could reliably tell multiple objects apart from one another and reach out to touch themsomething he couldnt do before. ECG readings also showed brain activity in the man linked to vision. Whats more, these improvements remained even five months after the initial treatment.

The mans restored vision was only possible while wearing the goggles, and the vision he did gain back was blurry and limited in range. But according to the authors, this is the first evidence in humans to definitively demonstrate that the therapy has potential to someday treat this rare but life-altering form of blindness.

In this study, we present the first evidence that injection of an optogenetic sensor-expressing gene therapy vector combined with the wearing of light-stimulating goggles can partially restore visual function in a patient, they wrote.

Other researchers are developing treatments for blindness using optogenetics, some with different approaches than the amber light translating goggles used in this study. Time will have to tell whether any of this research will ultimately pan out.But for now, hopes are high for good reason.

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Man Blinded for 40 Years Has Sight Partially Restored Using Gene Therapy - Gizmodo

Retinitis pigmentosa (RP) is the name given to a group of hereditary eye conditions called retinal dystrophy.

Retina dystrophy, such as RP, affects the retina behind the eye, and over time the retina becomes non-functional.

This means that RP causes gradual but permanent changes that reduce vision.

How much vision you lose, how quickly this happens, and the age at which it begins depends on the type of RP you have.

Changes in your eyesight occur over the years, not months, and some people lose their eyesight more than others.

What are the symptoms of RP?

With retinal dystrophy like RP, the rod and pyramidal cells of the eye gradually fail.

Depending on the type of RP you are using, you may notice the first symptoms between the ages of 10 and 30 after infancy.

Some people are asymptomatic until later in life.

With RP, the first symptom you notice is that you dont look like a person without vision in dimly lit places, such as at dusk or at night. This is often referred to as night blindness.

People without vision can fully adapt to dim light in 15-30 minutes, but when using RP it takes much longer or does not occur at all.

You may start to have trouble seeing things in the peripheral vision. You may miss things on either side of you, and you may trip over or bump into what you might have seen in the past.

As the RP progresses, peripheral vision is gradually lost, leaving a narrow central vision. This is often referred to as tunnel vision. You may still have a central vision until you are in your 50s, 60s, or older.

However, advanced RP often affects central vision, which can make it difficult to read and recognize the face.

RP is a progressive state. In other words, eyesight keeps getting worse year by year.

In many cases, changes in vision can occur suddenly in a short period of time. That way, you may get some vision for quite some time. However, the vision may change further in the future.

This may mean that you need to continue to re-adapt to lower levels of vision. The type of RP you are using can affect how quickly these changes occur.

What is the cause of RP?

RP is a hereditary disease caused by a defect in one of the genes involved in maintaining retinal health.

In RP, defective genes cause retinal cells to cease functioning and eventually die over time.

Researchers have identified many of the genes that cause RP and defects in it, but there are still other genes to discover.

Genetic testing

You can perform a genetic test to find out if there is a defective gene that causes RP.

This allows you to identify the defective gene that causes RP and to find out if your child has a defective gene that they may inherit.

A genetic test uses a blood test to look at a gene to see if it is defective.

Testing for RP and other hereditary retinal dystrophy is complex. Not all forms of these conditions are identified, as new defective genes are still being discovered.

Genetic counseling

Genetic counseling helps you understand the type of RP you have, how it may affect you in the future, and the risks of communicating that condition to the child you may have. Useful.

Genetic counseling is usually recommended when undergoing a genetic test. A genetic counselor will ask you more about your family tree and try to understand how RP is passed down to your family.

A genetic condition in your family can cause emotional concerns. Talking to a genetic counselor may help you and your family discuss your familys eye condition.

If you are thinking of starting a family, knowing the possibility of passing in any condition you have can help.

What tests are used to detect RP?

Optometrists (also known as opticians) can examine the retina to detect RP. If there are early signs of classic RP, there is a small but characteristic dark pigment mass around the retina.

Changes in peripheral vision can be detected by visual field testing. If your optometrist is worried after your eye exam, they can refer you to an ophthalmologist (hospital ophthalmologist) for further examination.

There are various tests that can diagnose RP. These tests also allow you to monitor how the RP changes over time.

Your ophthalmologist may be able to say that you have RP when you get the results of these tests, but the type of RP you have and the long term to your eyesight Tests that may not be known exactly if the effects are not genetic.

Some tests you may need to take include:

Source: Royal National Institute of Blind

Health: Blind (58 years old) partially recovered vision with pioneering gene therapy

Source link Health: Blind (58 years old) partially recovered vision with pioneering gene therapy

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Health: Blind (58 years old) partially recovered vision with pioneering gene therapy - Texasnewstoday.com

When kids struggle at school, parents and teachers try to identify the cause of the problem. They wonder whether the student can see or hear properly, or whether he or she has learning problems such as dyslexia. An ordinary eye test might show that a child can see perfectly well, but it may not tell the full story. Visual skills can be just as important as eyesight itself, and this point is often poorly understood or overlooked, says Gary Rodney, a behavioural optometrist and fellow of the International Academy of Orthokeratology and Myopia Control.

Being able to focus on an eye-test chart isnt enough. Its also important to be able to focus at various distances and change focus easily from one distance to another. Apart from being able to focus, eyes need to be able to track from one thing to the next. For example, when reading, the eyes must be able to scan from one word to the next without losing their place. At the same time, both eyes need to be able to work together as a team.

Over and above these basic visual skills, people need the ability to process spatial information, notice small differences between one thing and another, be able to coordinate what is done in relation to what is seen (eye-hand coordination), and be able to associate what is seen with what is heard.

Any issues with these visual skills will naturally result in problems with schoolwork. Up to 25 percent of children have visual difficulties of one kind or another, so having a thorough eye examination that includes testing of visual skills is a sensible step to take when children struggle at school.

Regular Optometrists and eye surgeons (Ophthalmologists) are trained to test how well one can see, but it takes further training to be able to comprehensively test all the visual skills needed to succeed at school.

Thats why many parents and educators eliminate issues with vision as a reason for children having difficulty with schoolwork too soon. After all, ordinary eye tests may show that a child has 20/20 vision, but without evaluation of the additional visual skills that will allow children to use their eyesight in learning, parents and teachers can mistake visual processing disorders for learning problems. As a result, the real issue is not addressed, and a child may be unable to fulfil his or her true potential.

Gary Rodney acknowledges that although full evaluation of vision and related visual skills can identify issues that are holding children back, other factors may contribute to difficulties with learning.

If a child is struggling at school, testing of visual and auditory skills are a good first step. Garys practice, Eyes in Design, located in Mosman, will also be able to refer parents to the relevant health practitioners if dyslexia or other learning problems are suspected but have not been diagnosed. Even when a formal diagnosis has occurred, addressing any visual skills difficulties will help children to deal better with the challenges they face.

Vision therapy is not a panacea, says Gary. However sometimes, its all thats needed to get kids on track. After all it has been shown that 80% of how we learn comes through the visual pathway. In other cases vision may not be the cause of the problem, or it may only be a contributing factor. Either way, Eyes in Designs vision therapists are ready to assist parents in finding the practitioners they need to help children with the learning challenges they face.

HEAR: Ultra106.5FM Interview with Gary Rodney Covid and Myopia: What you need to know!

For more information on vision therapy, or to book an appointment for a visual perception test online, visit the Smart Vision website:Optometrists Sydney: Optometry Services For Children and Adults | Smart Vision; for specific information about Myopia treatment and prevention visitMyopia Prevention: Solutions, Control And Treatment In Sydney; and for detailed information about Myopia Treatment visitOrthokeratology In Sydney: The Non Surgical Alternative.

To book an appointment for a thorough eye check-up,click hereor Call the Bondi clinic on (02) 9365 5047 or the Mosman clinic on (02) 9969 1600.

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Global Bionic Eye Market is valued at approximately USD 183.5 million in 2019 and is anticipated to grow with a healthy growth rate of more than 12.7% over the forecast period 2020-2027. Bionic eye, also referred to as visual prosthesis, is an experimental visual device used to restore the visual function of the patient experiencing partial or complete blindness. Many devices and surgeries have been developed, typically modeled on the cochlear implant or bionic ear device, so as to heal the blindness of the individuals.

Bionic eye is usually considered as artificial eye which can deliver visual sensation to the brain and often helps the people to improve the vision abilities, which is resulting in the higher market growth across the globe. Furthermore, the rise in prevalence of ophthalmic diseases due to growing geriatric population, increasing funding by public and private organizations, and intensive research and development (R&D) focused on product innovation are the few factors responsible for the impressive CAGR of the market during the forecast period. According to the World Health Organization, nearly 2.2 billion people were affected with vision impairment or blindness in October 2019, of which at least 1 billion people had preventable or unaddressed conditions around the world. Similarly, as per the International Agency for the Prevention of Blindness, in 2018, more than 21 million of Vietnamese required eye care services, Out of which over 3 million Vietnamese students are having eyesight problems. Thus, surge in vision-related complications may promote the adoption of the therapeutic or corrective devices, which has led to a boost in the demand for bionic eye all over the world. However, the high cost of bionic eye and the high research and development cost are the prime factors limiting the market growth over the forecast period of 2020-2027.

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The regional analysis of the global Bionic Eye market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America, and the Rest of the World. North America is the leading/significant region across the world in terms of market share owing to the rising awareness concerning vision-related problem, intensive research activities, and the presence of a significant number of market vendors in the region. Whereas Asia-Pacific is also anticipated to exhibit the highest growth rate / CAGR over the forecast period 2020-2027. Factors such as the rising prevalence eye-related or ophthalmic disorder such as myopia and presbyopia coupled with growing geriatric population across developing countries, such as China and India, would create lucrative growth prospects for the Bionic Eye market across the Asia-Pacific region.

Major market player included in this report are:Second Sight Medical Products LLCNidek Co. Ltd.Nano Retina Ltd.MetaModal LLCBiomedical TechnologiesBionic Vision TechnologiesNeoStrataMonash Vision GroupPixium Vision

The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players. The detailed segments and sub-segment of the market are explained below:

By Type:External EyeImplanted Eye

By Technology:ElectronicMechanical

By End-Use:HospitalsOphthalmic ClinicsOthers

By Region:North AmericaU.S.CanadaEuropeUKGermanyFranceSpainItalyROE

Asia PacificChinaIndiaJapanAustraliaSouth KoreaRoAPACLatin AmericaBrazilMexicoRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2017, 2018Base year 2019Forecast period 2020 to 2027

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Target Audience of the Global Bionic Eye Market in Market Study:

Key Consulting Companies & AdvisorsLarge, medium-sized, and small enterprisesVenture capitalistsValue-Added Resellers (VARs)Third-party knowledge providersInvestment bankersInvestors

Read more:
Bionic Eye Market Size Prognosticated to Perceive a Thriving Growth by 2027 interpreted by a new report The Courier - The Courier