StemCell Therapy

Like many medical facilities across the nation, our supply chain is feeling the effects of Hurricane Helenes aftermath. Johns Hopkins Medicine currently has a sufficient sterile fluid supply to meet treatment, surgical and emergency needs. However, we have put proactive conservation measures into place to ensure normal operations, always with patient safety as our first priority. Examples of sterile fluids include intravenous (IV), irrigation and dialysis fluids. Learn more.

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Specialty Clinics | Johns Hopkins Institute of Genetic Medicine

Like many medical facilities across the nation, our supply chain is feeling the effects of Hurricane Helenes aftermath. Johns Hopkins Medicine currently has a sufficient sterile fluid supply to meet treatment, surgical and emergency needs. However, we have put proactive conservation measures into place to ensure normal operations, always with patient safety as our first priority. Examples of sterile fluids include intravenous (IV), irrigation and dialysis fluids. Learn more.

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Pediatric Genetic Medicine at Johns Hopkins Children's Center

The Department of Genetic Medicine maintains several large centers funded in part by the National Institutes of Health. These research resources have a long history at Johns Hopkins and provide the foundation for innovative research, as well as providing services and information to scientists around the world.

OMIM, is an encyclopedia of genetic disorders, their clinical features and the genes that contribute to them. The database contains information on thousands ofMendelian conditions, disorders caused by errors in a single gene. The database was developed 55years ago by Victor McKusick and is now maintained byAda Hamosh, MD, MPH, and her team. OMIM is used by 2.7 million unique users per year around the world.

GRCFprovides year-roundresearch expertise, products, and services for the study of the human genome. At the leading edge of technology, the GRCF provides sophisticated tools and equipment oftentimes not available in individual research labs. The mission of the GRCF is to provide high quality, cost effective research services and products to investigators throughout the Johns Hopkins scientific community. Accordingly, GRCF services cover a broad segment of genetic research including:

JHG provides research and clinical genotyping and sequencing together with extensive analytic expertise. A partnership between the Departments of Genetic Medicineand Pathology, JHG opened its doors in 2017, co-localizing four existing labs:

CIDR,is a national resource, offering sequencing, genotyping and epigenetic services to scientists looking to discover genes and variants that contribute to human disease. As part of Johns Hopkins Genomics, CIDR researchers focus on the genetic architecture of complex traits, looking at conditions that result from many genetic variants and how these variants accumulate to manifest disease. This includes conditions such as all types of cancer risk, eye diseases, cleft lip and palate, oral health, environmental influences on child health outcomes, ADHD, structural brain disorders, obesity, alcoholism and aging. Most recent studies are focused on minority populations or extremely well-phenotyped populations. CIDR facilitates data cleaning and data sharing. The 140 CIDR studies posted in dbGaP are heavily utilized with > 7,600 data requests. Since opening its doors in 1996, CIDR has been continuously funded by contracts from a consortium of ten National Institutes of Health institutes (the CIDR Program) as well as through funding from many other genomic consortia, including most recently the national precision medicine initiative, the All of Us Research Program. As of January 2024, CIDR has completed 1,508 studies, consisting of > 1.7 million DNA samples and encompassing over 200 different phenotypes for 421 principal investigators world-wide.

BHCMGaccepts samples from thousands of peoplewith rare disorders submitted by a worldwide network of rare-disease experts. A collaboration between Baylor College of Medicine and Johns Hopkins, the goal of the center is to sequence the genomes of people with these conditions as well as appropriate family members to identify the genes and variants responsible for disorders whose molecular basis was previously unknown. In particular, the center seeks families with known or novel conditions for which the culprit gene is unknown. Successful identification of the responsible gene connects a particular gene with a particular set of clinical features, thereby enabling precise molecular diagnosis and prognosis.It alsoinforms research on the development of rational treatment and providing families with information about recurrence risk.

Focused on Kabuki syndrome and related Mendelian disorders of the epigenetic machinery. These rare disorders result from mutations in single genes encoding components of the systems that add, interpret or delete epigenetic marks with the result that sets of genes are mis-regulated. Currently we know of more than 40 such epigenetic disorders, most of which have intellectual disability and growth abnormalities as prominent clinical consequences. By understanding the features and pathogenesis of these precise abnormalities of the epigenetic system IGM investigators expect to understand not only each disorder but also to how the whole epigenetic systems functions and the pathophysiological consequences that accrue when the system malfunctions. This research complements the clinical services offered in the IGM Epigenetics and Chromatin Clinic where patients with these disorders are diagnosed, characterized and treated.

Focused on understanding the molecular pathophysiology of the vascular form of Ehlers-Danlos syndrome (vascular EDS) with the aim of providing informed management of these patients as well as developing new forms of therapy. The Center will utilize advanced genetic and molecular methods to discover the sequence of events that contribute to structural weakening of the arterial wall and internal tissues over time, ultimately leading to tear or rupture and the potential for early death. The research team has developed two mouse models of vascular EDS that demonstrate most of the important physical findings seen in patients with the disorder. As in people with vascular EDS, we observe tremendous variation in the timing of onset and severity of vascular disease in our mouse colonies. Our strong belief is that both genetic and environmental factors have the capacity to afford strong protection in vascular EDS. Once identified, we will attempt to mimic the mechanism of protection using medications or other strategies. The Center also aims to coordinate expert clinical care of individuals with vascular EDS, and to promote research in the clinical sciences that will improve both the length and quality of life for affected individuals. The Center for Vascular Ehlers-Danlos Syndrome Research has received generous and visionary funding from a variety of sources including the EDS Network CARES Foundation, the EDS Today Advocates, the DEFY Foundation, the Aldredge Family Foundation, and the Daskal Family Foundation.

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Research Centers | Johns Hopkins Institute of Genetic Medicine

The overall goal of the Department of Genetic Medicine is to integrate genetics into all of medicine. To this end, department of genetic medicine investigators are exploring the role of the genes and genetic variation in the generation of human phenotypes and using this knowledge in various ways to understand biology and to improve health.

The Department of Genetic Medicineclinical service is aimed at providing state-of-the-art care for our patients, as well as contributing to translational, patient-oriented research; providing a set of educational activities for our trainees; and importantly, serving as an exemplar of how genetics informs the care of individual patients. We recognize that for these activities to be successful we also must be active in the education of our students, our colleagues and the public at large.

The Department of Genetic Medicine has a committee on diversity, equity and inclusion. The committee's mission is to promote the personal and professional flourishing of individuals from all backgrounds, perspectives, and abilities. We seek to promote mutual respect and collaboration between individuals of diverse race, ethnicity, culture, physical characteristics, sex and gender identity, religion and nationality.

For Department members: read more on our Sharepoint Intranet site about committee members, events and initiatives.

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About Us - Johns Hopkins Medicine

Like many medical facilities across the nation, our supply chain is feeling the effects of Hurricane Helenes aftermath. Johns Hopkins Medicine currently has a sufficient sterile fluid supply to meet treatment, surgical and emergency needs. However, we have put proactive conservation measures into place to ensure normal operations, always with patient safety as our first priority. Examples of sterile fluids include intravenous (IV), irrigation and dialysis fluids. Learn more.

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Graduate Programs & Training | Johns Hopkins Medicine

To request an appointment in any of our genetics clinics, please call410-955-3071.

If this is your first visit to a genetics clinic at Johns Hopkins, the following steps will help you navigate making an appointment. From start to finish, scheduling an appointment may take up to 10 days, depending on the speed of insurance clearance, receiving records, and other factors.

Call our central appointment line at 410-955-3071. Our staff in the genetics office will walk you through the steps to making an appointment. They will collect general and insurance information about the patient and will send you a medical history questionnaire.

Complete one of the following medical history questionnaires, and fax the completed questionnaire to 410-367-3231.

Your primary care physician can help you complete the questionnaire. Genetic counselors review the questionnaire to determine each persons medical urgency and the appropriate medical providers to schedule the appointment. People with medical urgency who should receive appointments sooner than the general population of our patients include infants under six months of age, children whose physicians diagnosed them as failure to thrive or children who have lost developmental milestones.Generally, our next available appointments are four to six months from the time you first call our appointment line.

Our financial specialists will review your insurance information to confirm that it is active and will cover a visit with a medical geneticist, genetic counselor, dieticianand nurse. They will also help obtain referralsand will determine eligibility and coverage for genetic testing. You can help make this process faster by asking your primary care provider to fax a referral and records to 410-367-3231.

After your questionnaire and insurance status have been reviewed, our scheduling staff will contact you to schedule the first available appointment.

Questions about the status of your appointment?Call the main appointment line at 410-955-3071, Option 1

If you have been seen at one of our genetics clinics within three years, call 410-955-3071, option 2, to schedule your follow-up appointment.

If three or more years have passed since your last appointment at one of our genetics clinics, please follow the instructions for new patients.You will not need to submit a new medical questionnaire. The genetic counselors will review your genetic medical record.

Book follow-up visits early!Available appointments fill quickly, so dont delay in scheduling your next visit.

Johns Hopkins Medicine International pairs you with a medical concierge to arrange all aspects of your medical visit, paying special attention to your personal, cultural and travel-related needs. Your medical concierge can arrange consultations and treatment plans with the most appropriate specialists. Johns Hopkins Medicine International also provides language interpretation, financial counseling, assistance with travel arrangements and anything else to help make Johns Hopkins feel as close to home as possible.

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Request an Appointment | Johns Hopkins Institute of Genetic Medicine

Curt Medeiros on Revolutionizing Precision Medicine and Scaling Ovation  Madrona Venture Group

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Curt Medeiros on Revolutionizing Precision Medicine and Scaling Ovation - Madrona Venture Group

Catalysing ancient wisdom and modern science for the health and well-being of people and planet

Traditional medicine has been central to peoples health and well-being across cultures and countries for centuries. It has contributed to foundational medical texts and modern scientific breakthroughs. Landmark drugs like aspirin and artemisinin, as well as practices like yoga and meditation, originate from traditional medicine. Traditional medicine is at the frontiers of modern science and health care with advances in genomics and artificial intelligence, and personalized, holistic approaches. Increasing global attention is fueling related industries like bioeconomy and wellness valued at trillions of dollars.

Around 90% of WHO Member States have reported on the use of traditional medicine and requested robust evidence and data to guide policies, practice and regulations to ensure its safe and effective use, while promoting equity and sustainability.

To address this global demand, WHO established the Global Traditional Medicine Centre (GTMC) in 2022 with foundational support from the Government of India. The GTMC is a WHO Headquarters department, in the Division of Universal Health Coverage and Life Course, that is outposted to Jamnagar, Gujarat, India. The Centre focuses on advancing research, facilitating knowledge exchange, conserving biodiversity, and fostering partnerships to catalyze ancient wisdom and modern science for the health and well-being of people and planet.

The regional inputs are crucial for the draft WHO Global Strategy for Traditional Medicine 2025-2034 which is being formulated and the draft version...

The Fourth Forum was convened in Siem Reap on 2829 November 2022 in a hybrid format and brought together 216 participants from across the Western...

Siddha medicine is a popular health resource used across the world. Standard terminology relating to Siddha medicine is, therefore, an essential tool for...

Ayurveda is one of the popularly applied health resources across the globe. Standard terminology of Ayurveda is an essential tool for working on other...

The first WHO Traditional Medicine Global Summit Towards health and well-being for all was held in Gandhinagar, Gujarat, India, on 17-18...

Traditional and complementary medicine (T&CM) is an important and often underestimated health care resource. It has strong potential for preventing...

Following a high-level policy dialogue between the Director-General of WHO and Chinas National Administration of Traditional Chinese Medicine (NATCM)...

Get an overview of the WHOs Global Centre for Traditional Medicine (GCTM) initiative. Further explore the GCTM technical work streams and milestones,...

Multimedia

Traditional, complementary and integrative medicine in the WHO HQ and regions

African traditional medicine

Traditional Arab and Islamic medicine, including Unani

Ayurveda, yoga, unani, nuad Thai

Traditional Chinese medicine, acupuncture, tuina

Integrated Health Service Department

Related health topics

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WHO Global Traditional Medicine Centre

How to Identify and Prevent Frostbite  A Healthier Michigan

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How to Identify and Prevent Frostbite - A Healthier Michigan

Preventive care with Nutrition key to overcome heightened disease burden in India: Experts  ETHealthWorld

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Preventive care with Nutrition key to overcome heightened disease burden in India: Experts - ETHealthWorld

Alma Jacobsen, O.D. Family Eye Care & Contact Lens offers TAILORED VISUAL SOLUTIONS FOR TODAY'S UNIQUE VISUAL DEMANDS. Expect the best comprehensive eye care and targeted visual recommendations so you can see your best for life!Click or call to connect and access the quality eye care you deserve.

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Optometrist / Eye Doctor in North Brunswick and Hamilton, NJ - Alma ...

We are conveniently located in Somerset, New Jersey. We are easily accessible from North Brunswick and New Brunswick as well. Our doctors are here to take care of all your ophthalmologic needs. You can see any of our knowledgeable doctors: Call us today at (732) 745-4844 to make an appointment. Fax: (732) 545-3423.

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Shamim Eye Care of Central New Jersey

Cutaneous Carbonyl Stress Linked to Nerve Dysfunction and Neuropathy Risk in Recent-Onset Type 2...  Medical Dialogues

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Cutaneous Carbonyl Stress Linked to Nerve Dysfunction and Neuropathy Risk in Recent-Onset Type 2... - Medical Dialogues

Lack of association between common polymorphisms associated with successful aging and longevity in the population of Sardinian Blue Zone  Nature.com

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Lack of association between common polymorphisms associated with successful aging and longevity in the population of Sardinian Blue Zone - Nature.com

Scientists Unravel the Secrets of 37 Key Genes Linked to Reproductive Health and Longevity  SciTechDaily

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Scientists Unravel the Secrets of 37 Key Genes Linked to Reproductive Health and Longevity - SciTechDaily

Exosomes Are Being Hyped as a Silver Bullet Therapy. Scientists Say No.  Singularity Hub

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Exosomes Are Being Hyped as a Silver Bullet Therapy. Scientists Say No. - Singularity Hub

Using technology first designed by Bryan L. Roth, MD, PhD, the Michael Hooker Distinguished Professor of Pharmacology, researchers at the UNC School of Medicine have engineered a molecular technology that can turn off pain receptors.

Pain is meant to be a defense mechanism. It creates a strong sensation to get us to respond to a stimulus and prevent ourselves from further harm. But, sometimes injuries, nerve damage, or infections can cause long-lasting, severe bouts of pain that can make daily life unbearable.

What if there was a way to simply turn off pain receptors? UNC School of Medicine researchers Bryan L. Roth, MD, PhD, the Michael Hooker Distinguished Professor of Pharmacology, and Grgory Scherrer, PharmD, PhD, associate professor of cell biology and physiology and the UNC Neuroscience Center, have just proved that it is possible.

Using a tool designed by Roth in the early 2000s, the labs have created a new system that reduces acute and tissue-injury-induced inflammatory pain in mouse models. Hye Jin Kang, PhD, an alumnus of the Roth Lab and now associate professor at Yonsei University in Korea, was first author on the research paper. Their results were published in Cell.

What we have developed is potentially a gene therapy approach for chronic pain, said Roth, who is also a member of UNC Lineberger Comprehensive Cancer Center. The idea is that we could deliver this chemogenetic tool through a virus to the neurons that sense the pain. Then, you could just take an inert pill and turn those neurons off, and the pain will literally disappear.

Neuroscientists have been on a decades-long endeavor to build a comprehensive map of the human brain. If every type of cell and every neural pathway could be identified, researchers could make large strides in neurological research including the ability to turn regions of the brain on and off to parse out their functions or mimic drug therapy.

In the 90s, Roth, then professor of biochemistry at Case Western Reserve University (with secondary appointments in Psychiatry, Oncology, and Neurosciences), wanted to find a way to make new, powerful therapeutics that could stop diseases without incurring dissuading side effects. It was a tall order, pharmacologically-speaking. So, Roth decided to use an up-and-coming technique called directed molecular evolution, which essentially uses chemically engineered molecules to speed up the evolution process in nature.

What I realized, and what a lot of people realized, is, if you could make an engineered receptor that had some of the same signaling properties as a drug of interest, and if you could put it in a particular brain region or cell type, then you could mimic the effects of the drug, said Roth, who is now the project director of the NIMH Psychoactive Drug Screening Program. We made some several attempts in the 90s, as did other people, without a great deal of success.

Roth perfected the chemogenetic technology in 2005. With yeast as his model organism, he engineered an artificial protein receptor that could only be unlocked by clozapine N-oxide, a synthetic drug-like compound that had been rendered inert by removing all its therapeutic qualities.

The tool, which is also termed designer receptors exclusively activated by designer drugs, or DREADDs, acts as a molecular lock and key that can only be activated when an inert drug-like compound is introduced to the body. Once activated, the technology can turn neurons on or shut them off, effectively giving researchers the ability to make highly selective changes to the nervous system.

The techniques were revealed to the scientific community in March 2007 in the Proceedings of the National Academy of Sciences. Since then, Roths technology has been used by thousands of researchers worldwide to study the functions of neurons and develop new medications to treat complex neuropsychiatric conditions from depression and substance abuse to epilepsy and schizophrenia.

Every neuron in our body that is not part of central nervous system (CNS) belongs to the peripheral nervous system, or PNS. This division of the nervous system is responsible for relaying our five sensations to the CNS, allows our muscles to move, and aids in involuntary process such as digestion, breathing, and heart beats.

Relatively few studies have been done on the use of chemogenetics in the PNS, simply because of technical difficulty. The CNS and PNS are so intertwined on a cellular, chemical, and genetic level, that it is challenging for researchers to apply their technology solely to the PNS.

Many of the genes that are expressed in the peripheral nervous system are also expressed in the central nervous system, particularly in the brain, said Scherrer, who is also an associate professor in the UNC Department of Pharmacology. We had to perform a multitude of analyses and tests to isolate both a receptor and drug-like compound that only operate in the periphery.

However, after seven long years, the Roth and Scherrer labs found success. Researchers based their new system off of hydroxycarboxylic acid receptor 2 (HCA2), a type of receptor implicated in anti-inflammation. HCA2 receptors are expressed in the PNS and are usually activated by vitamin B3. Using mouse models, researchers altered the HCA2 receptors so that they could only bind to FCH-2296413, an inert drug-like compound that only acts within the PNS.

The chemogenetic system, termed mHCAD, is designed to interfere with nociceptors, making it more difficult for the sensory neurons to transmit pain information to the spinal cord and brain. To be more specific, mHCAD reduces their ability to fire off their electrical and chemical messages. A more intense, more painful stimulus will be needed to cause the perception of pain.

Although the technology is still far from human use, Roth and Scherrer have already thought about how the technology would best be delivered in the body: through gene therapy. Researchers successfully injected mHCAD into a mouse model using genetic technology created by colleague and gene therapy pioneer Jude Samulski, PhD, a distinguished professor of pharmacology at the UNC School of Medicine. The gene therapy leverages the infectious abilities of the adeno-associated virus (AAV), allowing researchers to deliver mHCAD into the pain neurons of interest.

In 2013, the National Institutes of Health formed a partnership between Federal and non-Federal partners with a common goal of mapping every human brain cell and every neural circuit through innovative neurotechnologies called the Brain Research Through Advancing Innovative Neurotechnologies Initiative, or BRAIN Initiative.

Roths chemogenetic technology has played a big role in the BRAIN Initiative. To date, tens of thousands of shipments of viruses and plasmids from the Roth lab have been distributed leading to many thousand publications. Now that the technology has expanded to the peripheral nervous system, researchers can better study the neurons that produce the perception of touch, temperature, body position, pain, and more.

There are dozens of classes of PNS neurons that we dont fully understand, said Scherrer. By using this new innovative tool, we can then define cellular targets that we can engage with to treat diseases. Its going to be an important tool to increase our knowledge in the somatosensory field and beyond.

Media contact:Kendall Daniels Rovinsky, Communications Specialist, UNC Health | UNC School of Medicine

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Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ...

July 27, 2020, by NCI Staff

CRISPR is a highly precise gene editing tool that is changing cancer research and treatment.

Credit: Ernesto del Aguila III, National Human Genome Research Institute

Ever since scientists realized that changes in DNA cause cancer, they have been searching for an easy way to correct those changes by manipulating DNA. Although several methods of gene editing have been developed over the years, none has really fit the bill for a quick, easy, and cheap technology.

But a game-changer occurred in 2013, when several researchers showed that a gene-editing tool called CRISPR could alter the DNA of human cells like a very precise and easy-to-use pair of scissors.

The new tool has taken the research world by storm, markedly shifting the line between possible and impossible. As soon as CRISPR made its way onto the shelves and freezers of labs around the world, cancer researchers jumped at the chance to use it.

CRISPR is becoming a mainstream methodology used in many cancer biology studies because of the convenience of the technique, said Jerry Li, M.D., Ph.D., of NCIs Division of Cancer Biology.

Now CRISPR is moving out of lab dishes and into trials of people with cancer. In a small study, for example, researchers tested a cancer treatment involving immune cells that were CRISPR-edited to better hunt down and attack cancer.

Despite all the excitement, scientists have been proceeding cautiously, feeling out the tools strengths and pitfalls, setting best practices, and debating the social and ethical consequences of gene editing in humans.

Like many other advances in science and medicine, CRISPR was inspired by nature. In this case, the idea was borrowed from a simple defense mechanism found in some microbes, such as bacteria.

To protect themselves against invaders like viruses, these microbes capture snippets of the intruders DNA and store them away as segments called CRISPRs, or clustered regularly interspersed short palindromic repeats. If the same germ tries to attack again, those DNA segments (turned into short pieces of RNA) help an enzyme called Cas find and slice up the invaders DNA.

After this defense system was discovered, scientists realized that it had the makings of a versatile gene-editing tool. Within a handful of years, multiple groups had successfully adapted the system to edit virtually any section of DNA, first in the cells of other microbes, and then eventually in human cells.

CRISPR consists of a guide RNA (RNA-targeting device, purple) and the Cas enzyme (blue). When the guide RNA matches up with the target DNA (orange), Cas cuts the DNA. A new segment of DNA (green) can then be added.

Credit: National Institute of General Medical Sciences, National Institutes of Health

In the laboratory, the CRISPR tool consists of two main actors: a guide RNA and a DNA-cutting enzyme, most commonly one called Cas9. Scientists design the guide RNA to mirror the DNA of the gene to be edited (called the target). The guide RNA partners with Cas andtrue to its nameleads Cas to the target. When the guide RNA matches up with the target gene's DNA, Cas cuts the DNA.

What happens next depends on the type of CRISPR tool thats being used. In some cases, the target gene's DNA is scrambled while it's repaired, and the gene is inactivated. With other versions of CRISPR, scientists can manipulate genes in more precise ways such as adding a new segment of DNA or editing single DNA letters.

Scientists have also used CRISPR to detect specific targets, such as DNA from cancer-causing viruses and RNA from cancer cells. Most recently, CRISPR has been put to use as an experimental testto detect the novel coronavirus.

Scientists consider CRISPR to be a game-changer for a number of reasons. Perhaps the biggest is that CRISPR is easy to use, especially compared with older gene-editing tools.

Before, only a handful of labs in the world could make the proper tools [for gene editing]. Now, even a high school student can make a change in a complex genome using CRISPR, said Alejandro Chavez, M.D., Ph.D., an assistant professor at Columbia University who has developed several novel CRISPR tools.

CRISPR is also completely customizable. It can edit virtually any segment of DNA within the 3 billion letters of the human genome, and its more precise than other DNA-editing tools.

And gene editing with CRISPR is a lot faster. With older methods, it usually [took] a year or two to generate a genetically engineered mouse model, if youre lucky, said Dr. Li. But now with CRISPR, a scientist can create a complex mouse model within a few months, he said.

Another plus is that CRISPR can be easily scaled up. Researchers can use hundreds of guide RNAs to manipulate and evaluate hundreds or thousands of genes at a time. Cancer researchers often use this type of experiment to pick out genes that might make good drug targets.

And as an added bonus, its certainly cheaper than previous methods, Dr. Chavez noted.

With all of its advantages over other gene-editing tools, CRISPR has become a go-to for scientists studying cancer. Theres also hope that it will have a place in treating cancer, too. But CRISPR isnt perfect, and its downsides have made many scientists cautious about its use in people.

A major pitfall is that CRISPR sometimes cuts DNA outside of the target genewhats known as off-target editing. Scientists are worried that such unintended edits could be harmful and could even turn cells cancerous, as occurred in a 2002 study of a gene therapy.

If [CRISPR] starts breaking random parts of the genome, the cell can start stitching things together in really weird ways, and theres some concern about that becoming cancer, Dr. Chavez explained. But by tweaking the structures of Cas and the guide RNA, scientists have improved CRISPRs ability to cut only the intended target, he added.

Another potential roadblock is getting CRISPR components into cells. The most common way to do this is to co-opt a virus to do the job. Instead of ferrying genes that cause disease, the virus is modified to carry genes for the guide RNA and Cas.

Slipping CRISPR into lab-grown cells is one thing; but getting it into cells in a person's bodyis another story. Some viruses used to carry CRISPR can infect multiple types of cells, so, for instance, they may end up editing muscle cells when the goal was to edit liver cells.

Researchers are exploring different ways to fine-tune the delivery of CRISPR to specific organs or cells in the human body. Some are testing viruses that infect only one organ, like the liver or brain. Others have created tiny structures callednanocapsules that are designed to deliver CRISPR components to specific cells.

Because CRISPR is just beginning to be tested in humans, there are also concerns about how the bodyin particular, the immune systemwill react to viruses carrying CRISPR or to the CRISPR components themselves.

Some wonder whether the immune system could attack Cas (a bacterial enzyme that is foreign to human bodies) and destroy CRISPR-edited cells. Twenty years ago, a patient died after his immune system launched a massive attack against the viruses carrying a gene therapy he had received. However, newer CRISPR-based approaches rely on viruses that appear to be safer than those used for older gene therapies.

Another major concern is that editing cells inside the body could accidentally make changes to sperm or egg cells that can be passed on to future generations. But for almost all ongoing human studies involving CRISPR, patients cells are removed and edited outside of their bodies. This ex vivo approach is considered safer because it is more controlled than trying to edit cells inside the body, Dr. Chavez said.

However, one ongoing study is testing CRISPR gene editing directly in the eyes of people with a genetic disease that causes blindness, called Leber congenital amaurosis.

The first trial in the United States to test a CRISPR-made cancer therapy was launched in 2019 at the University of Pennsylvania. The study, funded in part by NCI, is testing a type of immunotherapy in which patients own immune cells are genetically modified to better see and kill their cancer.

The therapy involves making four genetic modifications to T cells, immune cells that can kill cancer. First, the addition of a synthetic gene gives the T cells a claw-like protein (called a receptor) that sees NY-ESO-1, a molecule on some cancer cells.

Then CRISPR is used to remove three genes: two that can interfere with the NY-ESO-1 receptor and another that limits the cells cancer-killing abilities. The finished product, dubbed NYCE T cells, were grown in large numbers and then infused into patients.

The first trial of CRISPR for patients with cancer tested T cells that were modified to better "see" and kill cancer.CRISPR was used to remove three genes: two that can interfere with the NY-ESO-1 receptor and another that limits the cells cancer-killing abilities.

Credit: National Cancer Institute

We had done a prior study of NY-ESO-1directed T cells and saw some evidence of improved response and low toxicity, said the trials leader, Edward Stadtmauer, M.D., of the University of Pennsylvania. He and his colleagues wanted to see if removing the three genes with CRISPR would make the T cells work even better, he said.

The goal of this study was to first find out if the CRISPR-made treatment was safe. It was tested in two patients with advanced multiple myeloma and one with metastatic sarcoma. All three had tumors that contained NY-ESO-1, the target of the T-cell therapy.

Initial findings suggest that the treatment is safe. Some side effects did occur, but they were likely caused by the chemotherapy patients received before the infusion of NYCE cells, the researchers reported. There was no evidence of an immune reaction to the CRISPR-edited cells.

Only about 10% of the T cells used for the therapy had all four of the desired genetic edits. And off-target edits were found in the modified cells of all three patients. However, none of the cells with off-target edits grew in a way that suggested they had become cancer, Dr. Stadtmauer noted.

The treatment had a small effect on the patients cancers. The tumors of two patients (one with multiple myeloma and one with sarcoma) stopped growing for a while but resumed growing later. The treatment didn't work at all for the third patient.

It'sexciting that the treatment initially worked for the sarcoma patientbecause solid tumors have been a much more difficult nut to crack with cellular therapy," Dr. Stadtmauer said. "Perhaps [CRISPR] techniques will enhance our ability to treat solid tumors with cell therapies.

Although the trial shows that CRISPR-edited cell therapy is possible, the long-term effects still need to be monitored, Dr. Stadtmauer continued. The NYCE cells are safe for as long as weve been watching [the study participants]. Our plan is to keep monitoring them for years, if not decades, he said.

While the study of NYCE T cells marked the first trial of a CRISPR-based cancer treatment, there are likely more to come.

This [trial] was really a proof-of-principle, feasibility, and safety thing that now opens up the whole world of CRISPR editing and other techniques of [gene] editing to hopefully make the next generation of therapies, Dr. Stadtmauer said.

Other clinical studies of CRISPR-made cancer treatments are already underway. A few trials are testing CRISPR-engineered CAR T-cell therapies, another type of immunotherapy. For example, one company is testing CRISPR-engineered CAR T cells in people with B cell cancers and people with multiple myeloma.

There are still a lot of questions about all the ways that CRISPR might be put to use in cancer research and treatment. But one thing is for certain: The field is moving incredibly fast and new applications of the technology are constantly popping up.

People are still improving CRISPR methods, Dr. Li said. Its quite an active area of research and development. Im sure that CRISPR will have even broader applications in the future.

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How CRISPR Is Changing Cancer Research and Treatment

Patients with Leber hereditary optic neuropathy (LHON) due to the MT-ND4 gene variant treated with the gene therapy lenadogene nolparvovec in one eye showed a sustained improvement in best-corrected visual acuity (BCVA) in both eyes up to 5 years after treatment, the long-term RESTORE study showed.

Among 55 patients who completed the 5-year follow-up, bilateral vision improvement was similar to what was observed at the 2-year mark, with a mean change in BCVA of -0.4 logMAR (more than +4 lines) for treated eyes and -0.4 logMAR (+4 lines) for eyes treated with sham (difference -0.05, 95% CI -0.15 to 0.04, P=0.27), reported Patrick Yu-Wai-Man, MD, PhD, of the University of Cambridge in England, and colleagues.

In addition, an improvement of at least -0.3 logMAR (+3 lines) from the nadir in at least one eye was observed in 66.1% of patients, they noted in JAMA Ophthalmology.

The findings suggest that vision improvement "is likely to be a lasting effect," co-author Jos-Alain Sahel, MD, PhD, of the Centre Hospitalier National D'Ophtalmologie des Quinze Vingts in Paris, told MedPage Today. However, Sahel -- a co-founder of the company that makes the gene therapy -- said it may still take several years to get the treatment approved in the U.S.

LHON is a rare inherited disease that damages the optic nerve, and is estimated to affect one in 50,000 people. According to Sahel, patients with the condition have normal vision early in life but typically develop problems as teenagers or young adults. One eye and then the other loses vision, he said, and most patients become legally blind.

Sahel explained that lenadogene nolparvovec, an adeno-associated virus (AAV)-based ocular gene therapy, was designed to correct the genetic defect that causes LHON.

The initial RESCUE and REVERSE trials, patients from which comprise RESTORE, showed that vision in treated eyes improved at 2 years, but, unexpectedly, so did vision in untreated(sham) eyes, said Sahel, who noted that about 60% to 70% of patients were able to regain some level of vision.

"Most of them didn't regain full vision, but several of them regained a good level of autonomy and the ability to read at least large print or medium print," he added. Some patients were able to drive again, take part in sports, and go back to college.

This follow-up study showed that vision benefits persisted over 3 years beyond the initial studies, Sahel concluded, noting that no patients had permanent complications from the gene therapy, and inflammation occurring in some patients was manageable.

In an accompanying commentary, Hendrik Scholl, MD, of Medical University of Vienna, and Bence Gyrgy, MD, PhD, of the University of Basel in Switzerland, said that the initial results of the RESCUE and REVERSE trials were "somewhat puzzling."

The studies didn't meet their primary outcomes -- a difference in visual acuity between treated and untreated eyes -- since vision in both eyes improved, they wrote. "One possibility [for this] is that the vector is trafficking from one eye to the other."

Another possibility is the analysis may be misleading because the authors chose to track visual changes from nadir, which "may represent a 1-time event due to poor effort or poor fixation."

The commentary authors recommended that "if investigators insist on using change from nadir, at a minimum, they should require 2 consecutive visits that confirm the stability of this nadir visit and, more importantly and primarily, consider BCVA changes from baseline."

The RESCUE and REVERSE Long-Term Follow-up Study (RESTORE) was conducted from 2018 to 2022 and tracked 62 of the 76 patients with LHON due to the MT-ND4 gene variant who took part in the initial two trials; 55 patients completed the 5-year follow-up. Mean age at treatment was 35.9, and 79% were men.

The mean baseline BCVA was 1.5 logMAR in the lenadogene nolparvovec group and 1.4 logMAR in sham eyes; after 2 years, the mean changes were -0.05 logMAR (+1 line) and 0.01 logMAR (-0 line), respectively (difference -0.03, 95% CI -0.16 to 0.09, P=0.60).

Over the 3-year follow-up period, intraocular inflammation occurred in four patients with eight events in eyes treated with lenadogene nolparvovec and one event in an eye treated with sham.

Sahel acknowledged that the gene therapy is likely to be expensive. In 2021, its manufacturer said the bilateral treatment would cost $725,000 per patient.

What's next? "Most likely in the U.S. and in some countries in Europe, there will be a need for a confirmatory study to be performed," he said, in light of the unusual initial results that didn't allow comparison of treated and untreated eyes.

The company plans further studies, he added, and he expects approval in the U.S. to come in 3 to 4 years.

Randy Dotinga is a freelance medical and science journalist based in San Diego.

Disclosures

GenSight Biologics, maker of lenadogene nolparvovec, funded the study, with support from multiple sources including the U.K. National Institute of Health Research, U.K. Medical Research Council, Fight for Sight, Isaac Newton Trust, Moorfields Eye Charity, Addenbrooke's Charitable Trust, and others.

Yu-Wai-Man reported receiving consultant fees from GenSight Biologics, Chiesi, and Neurophth, and research support from GenSight Biologics and Santhera.

Sahel reported being a co-founder and shareholder of GenSight Biologics; receiving grants from Laboratoire d'Excellence (LabEx) Lifesenses, Institut Hospitalo-Universitaire FOReSIGHT, the NIH, Research to Prevent Blindness, Light4Deaf, and the European Research Council; having a patent related to gene therapy for LHON; a personal financial interest in Pixium, GenSight Biologics, Sparing Vision, Prophesee, Chronolife, Tilak, VegaVect, Avista, Tenpoint, and SharpEye; receiving consultant fees from Tenpoint and Avista; and serving on the board of GenSight and Sparing Vision.

Other study authors reported multiple and various disclosures, including relationships with GenSight Biologics.

Scholl reported being chief medical officer of Belite Bio and relationships with the Swiss National Science Foundation, Wellcome Trust, Droia, Janssen (Johnson & Johnson), Okuvision, Boehringer Ingelheim, Alnylam, Belite Bio, F. Hoffmann-La Roche, ViGeneron, and Novo Nordisk.

Gyrgy reported relationships with Sphere, Cove, and the Swiss National Science Foundation.

Primary Source

JAMA Ophthalmology

Source Reference: Yu-Wai-Man P, et al "Five-year outcomes of lenadogene nolparvovec gene therapy in Leber hereditary optic neuropathy" JAMA Ophthalmol 2024; DOI: 10.1001/jamaophthalmol.2024.5375.

Secondary Source

JAMA Ophthalmology

Source Reference: Scholl HPN, Gyrgy B "Single-eye gene therapy for Leber hereditary optic neuropathy" JAMA Ophthalmol 2024; DOI: 10.1001/jamaophthalmol.2024.5618.

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Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease

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100 cell and gene therapy leaders to watch in 2025