StemCell Therapy

On August 2nd, scientists achieved a milestone on the path to human genetic engineering. For the first time in the United States, scientists successfully edited the genes of a human embryo. A transpacific team of researchers used CRISPR-Cas9 to correct a mutation that leads to an often devastating heart condition. Responses to this feat followed well-trodden trails. Hype over designer babies. Hope over new tools to cure and curb disease. Some spin, some substance and a good dose of science-speak. But for me, this breakthrough is not just about science or medicine or the future of humankind. Its about faith and family, love and loss. Most of all, its about the life and memory of my brother.

Jason was born with muscle-eye-brain disease. In his case, this included muscular dystrophy, cerebral palsy, severe nearsightedness, hydrocephalus and intellectual disability. He lived past his first year thanks to marvels of modern medicine. A shunt surgery to drain excess cerebrospinal fluid building up around his brain took six attempts, but the seventh succeeded. Aside from those surgeries complications and intermittent illnesses due to a less-than-robust immune system, Jason was healthy. Healthy and happy very happy. His smile could light up a room. Yet, that didnt stop people from thinking that his disability made him worse off. My family and those in our religious community prayed for Jason. Strangers regularly came up to test their fervor. Prayer circles frequently had his name on their lists. We wanted him to be healed. But I now wonder: What, precisely, were we praying for?

Jasons disabilities fundamentally shaped his experience of the world. If praying for his healing meant praying for him to be normal, we were praying for Jason to become someone else entirely. We were praying for a paradox. If I could travel back in time, Id walk up to young, devout Joel and ask: How will Jason still be Jason if God flips a switch and makes him walk and talk and think like you? The answer to that question is hard. Yes, some just prayed for his seizures to stop. Some for his continued well-being. But is that true of most? Is that what I was praying for?

The ableist conflation of disability with disease and suffering is age-old. Just peruse the history of medicine. Decades of eugenic practices. Sanctioned torture of people with intellectual disability. The mutilation of otherwise healthy bodies in the name of functional or aesthetic normality. These stories demonstrate over and over again how easily biomedical research and practice can mask atrocity with benevolence and injustice with progress. Which leads me to ask: What, precisely, are we editing for?

Although muscle-eye-brain disease does not result from a single genetic variant, researchers agree that a single gene, named POMGNT1, plays a large role. Perhaps scientists will soon find a way to correct mutations in that and related genes. Perhaps people will no longer be born with it. But that means there would never be someone like Jason. Those prayers I mentioned above? Science will have retroactively answered them. That thought brings me to tears.

I wish we could cure cancer, relieve undue pain and heal each break and bruise. But I also wish for a world with Jason and people like him in it. I want a world accessible and habitable for people full stop not just the people we design. I worry that in our haste to make people healthy, we are in fact making people we want. We, who say we pray for healing, but in fact pray for others to be like us. We, who say were for reducing disease and promoting health, but support policies and practices aimed instead at being normal. We, who are often still unable to distinguish between positive, world-creating forms of disability and negative, world-destroying forms between Deafness, short stature or certain types of neurodiversity and chronic pain, Tay-Sachs or Alzheimers. It is with great responsibility that we as a society balance along the tightrope of biomedical progress. I long for us to find that balance. Ive certainly not found it for myself. Lest I forget how often weve lost it and how easy it is to fall, I hold dearly onto the living memory of Jason. I no longer pray for paradoxes, but for parity for the promise of a world engineered not for normality, but equality.

But that world will never come if we edit it away.

Go here to see the original:
Gene Editing Might Mean My Brother Would've Never Existed - TIME

The chip has not been tested in humans, but it has been used to heal wounds in mice. Wexner Medical Center/The Ohio State University hide caption

The chip has not been tested in humans, but it has been used to heal wounds in mice.

Scientists have created an electronic wafer that reprogrammed damaged skin cells on a mouse's leg to grow new blood vessels and help a wound heal.

One day, creator Chandan Sen hopes, it could be used to be used to treat wounds on humans. But that day is a long way off as are many other regeneration technologies in the works. Like Sen, some scientists have begun trying to directly reprogram one cell type into another for healing, while others are attempting to build organs or tissues from stem cells and organ-shaped scaffolding.

But other scientists have greeted Sen's mouse experiment, published in Nature Nanotechnology on Monday, with extreme skepticism. "My impression is that there's a lot of hyperbole here," says Sean Morrison, a stem cell researcher at the University of Texas Southwestern Medical Center. "The idea you can [reprogram] a limited number of cells in the skin and improve blood flow to an entire limb I think it's a pretty fantastic claim. I find it hard to believe."

When the device is placed on live skin and activated, it sends a small electrical pulse onto the skin cells' membrane, which opens a tiny window on the cell surface. "It's about 2 percent of the cell membrane," says Sen, who is a researcher in regenerative medicine at Ohio State University. Then, using a microscopic chute, the chip shoots new genetic code through that window and into the cell where it can begin reprogramming the cell for a new fate.

Sen says the whole process takes less than 0.1 seconds and can reprogram the cells resting underneath the device, which is about the size of a big toenail. The best part is that it's able to successfully deliver its genetic payload almost 100 percent of the time, he says. "No other gene delivery technique can deliver over 98 percent efficiency. That is our triumph."

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice. Wexner Medical Center/The Ohio State University hide caption

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice.

To test the device's healing capabilities, Sen and his colleagues took a few mice with damaged leg arteries and placed the chip on the skin near the damaged artery. That reprogrammed a centimeter or two of skin to turn into blood vessel cells. Sen says the cells that received the reprogramming genes actually started replicating the reprogramming code that the researchers originally inserted in the chip, repackaging it and sending it out to other nearby cells. And that initiated the growth of a new network of blood vessels in the leg that replaced the function of the original, damaged artery, the researchers say. "Not only did we make new cells, but those cells reorganized to make functional blood vessels that plumb with the existing vasculature and carry blood," Sen says. That was enough for the leg to fully recover. Injured mice that didn't get the chip never healed.

When the researchers used the chip on healthy legs, no new blood vessels formed. Sen says because injured mouse legs were was able to incorporate the chip's reprogramming code into the ongoing attempt to heal.

That idea hasn't quite been accepted by other researchers, however. "It's just a hand waving argument," Morrison says. "It could be true, but there's no evidence that reprogramming works differently in an injured tissue versus a non-injured tissue."

What's more, the role of exosomes, the vesicles that supposedly transmit the reprogramming command to other cells, has been contentious in medical science. "There are all manners of claims of these vesicles. It's not clear what these things are, and if it's a real biological process or if it's debris," Morrison says. "In my lab, we would want to do a lot more characterization of these exosomes before we make any claims like this."

Sen says that the theory that introduced reprogramming code from the chip or any other gene delivery method does need more work, but he isn't deterred by the criticism. "This clearly is a new conceptual development, and skepticism is understandable," he says. But he is steadfast in his confidence about the role of reprogrammed exosomes. When the researchers extracted the vesicles and injected them into skin cells in the lab, Sen says those cells converted into blood vessel cells in the petri dish. "I believe this is definitive evidence supporting that [these exosomes] may induce cell conversion."

Even if the device works as well as Sen and his colleagues hope it does, they only tested it on mice. Repairing deeper injuries, like vital organ damage, would also require inserting the chip into the body to reach the wound site. It has a long way to go before it can ever be considered for use on humans. Right now, scientists can only directly reprogram adult cells into a limited selection of other cell types like muscle, neurons and blood vessel cells. It'll be many years before scientists understand how to reprogram one cell type to become part of any of our other, many tissues.

Still, Morrison says the chip is an interesting bit of technology. "It's a cool idea, being able to release [genetic code] through nano channels," he says. "There may be applications where that's advantageous in some way in the future."

The rest is here:
A Chip That Reprograms Cells Helps Healing, At Least In Mice - NPR

The father of a boy with a rare genetic mutation has accused a scientist of exploiting his child by proclaiming the defect a genetic syndrome and naming it after herself.

At an impasse with scientists investigating, publicizing, and interpreting his sons condition, the father seems willing to use any leverage he can muster to remove the syndrome entry in an online genetic disease database. Based solely on an email he obtained from the database director, the father became convinced that if the paper underpinning the entry were retracted, the syndrome would go down with it. So earlier this year, he withdrew his consent and asked the journal that published the paper for a retraction, based on improper patient consent. He has also threatened to lob accusations of research misconduct at the papers last author.

Marc Pieterse, of The Netherlands, is the father of Vincent, a teenager who has a mutation in the RPS23 gene that has only been found in one other person, so far. In March, an international team of researchers published a paper on Vincents RPS23 mutation in the American Journal of Human Genetics (AJHG), linking it to defective ribosomes, organelles involved in protein synthesis.

One of the scientists Pieterse engaged several years ago is Alyson MacInnes, a rare disease researcher at the University of Amsterdams Academic Medical Center. She is last author of the AJHG paper and the person whose name is now connected to an entry in the Online Mendelian Inheritance in Man (OMIM) database. MacInnes told Retraction Watch that, contrary to what Pieterse claims, she played no direct role in naming the syndrome; OMIM confirmed this account.

The OMIM entry for MacInnes Syndrome, which links the RPS23 mutation with a collection of features that resemble Vincents hearing loss, issues with the hands was created on March 29, weeks after the paper was published. Pieterse said he was shocked when he found it in April as he was browsing the database.

Pieterse told us he feels used and fears that the designation will stigmatize his sons mutation. A syndrome is a disease, he said. Now, he wants the database entry either changed he prefers the umbrella term ribosomopathy, which is used in the paper or taken down.

Believing MacInnes submitted Vincents condition for consideration, Pieterse demanded she find a way to remove it. When she didnt respond, he went directly to AJHG and OMIM to get the paper and syndrome entry removed.

So far, nothing has worked.

A campaign begins

The Pieterses found out about Vincents mutation after a long diagnostic odyssey that ultimately resorted to sequencing all the protein-coding regions of Vincents genome. In 2015, the Journal of the American Medical Association published a news feature on Vincents diagnosis, saying it heralded a new era of clinical genomics.

Marc is a former telecommunications engineer and entrepreneur who has shifted his focus to raising his four children. He told Retraction Watch that although hes not a scientist, in the years since receiving Vincents diagnosis he has committed himself to advocating for further study of the mutation and has even co-authored a paper on RPS23. Marc claims he played a role in connecting MacInnes, Baserga, and several other European scientists, who eventually published the AJHG paper together.

When Pieterse found the OMIM entry for MacInnes syndrome, he believed that MacInnes had created it to boost her career. He told us that after he found it, he tried asking her to take it down. However, their relationship had at that point already suffered a communication breakdown and he didnt hear back. This further upset him and he began a campaign to bring down the entry by any means possible.

But MacInnes told us she had nothing to do with either the OMIM entrys creation or its naming:

I did not submit this paper to OMIM or in any way initiate this entry as a syndrome. This was independently picked up by OMIM and registered as such; apparently such registrations are made upon their decision only.

OMIM director Ada Hamosh confirmed this to Retraction Watch:

Dr. Macinnes did not ask for this to be named after herself and did not bring it to our attention.

We are dealing with this gene-phenotype relationship exactly as we would any other. We did this because this is what we do.

Hamosh, a geneticist at Johns Hopkins University, told us that the term syndrome is for a constellation of features and that the naming was done in accordance with policies that have long been in place at OMIM:

Sometimes something has too many features to be described succinctly. In that case, the default way to name something is to use the first authors last name and last authors last name.

Indeed, Hamosh told us that at first the syndrome was called Paolini-MacInnes syndrome, after first author Nahuel Paolini, of the University of Amsterdam. However, Hamosh said OMIM later realized there were four co-first authors. OMIM never adds more than three names to a syndrome, so Hamosh simply named it after MacInnes:

Given how little we know about it, it makes more sense to name it eponymously than after some features I cant put my hands on, especially since we have a policy on not ever naming something after a gene.

Its stigmatizing

Part of Pieterses issue with dubbing the condition a new syndrome is the early and ongoing nature of RPS23 research, and he isnt alone. In an email to Hamosh, MacInnes co-author Susan Baserga, a professor at the Yale School of Medicine, said:

I was very surprised that you are so pressed to name the phenotype as a new syndrome, especially since the clinical findings are so non-specific. I find this very odd indeed, and worry that it muddles the medical and genetic literature instead of providing clarity. This is so new that I am not even sure that it is a syndrome, and worry that it is presumptuous at best and wrong at worst.

Baserga, who did not respond to our requests for comment, also suggested that OMIM simply call the condition a ribosomopathy, as the AJHG paper does. But Hamosh told Retraction Watch:

We never, ever, ever, name a disease after a gene.

Gene symbols are not stable. More fundamentally, many, many, many genes have more than one condition associated with them. It is not a good idea to put a gene name into a disease name. Thats why we wont call it RPS23 ribosomopathy. Its not personal, we wont do this for any gene.

Pieterse told us that neither Hamosh, nor anybody else from OMIM, has ever informed him that OMIM itself created the entry and that MacInnes Syndrome is the result of standard naming procedures.

Like MacInnes, Hamosh wont respond to his attempt at contact. But Pieterse has obtained an email chain, from late April, between those two scientists, as well as Baserga. In it, Hamosh wrote:

Are you planning to retract or correct the paper to indicate the apparent uncertainty regarding its conclusions? If so, we will remove the phenotype and reclassify the variants.

Niether MacInnes nor Baserga thought a retraction was necessary, but this exchange convinced Pieterse that a retraction would force OMIM to remove the entry. So he wrote MacInnes to inform her he was withdrawing his parental consent and asked AJHG to retract the paper. Pieterse told Retraction Watch that the consent form he submitted to the University of Freiburgs medical center, in Germany (cells used in the study were created there) was very broad and that he believed it would allow him get the paper pulled.

Readers may recall some of the cases weve covered in which patient consent issues have led to papers being retracted. Pieterses situation most closely resembles a story we covered in 2015, where the authors requested a retraction from the Journal of Medical Case Reports after a legal guardian withdrew permission after publication.

But his attempt to trigger retraction didnt work. AJHG editor David Nelson, of the Baylor College of Medicine, told Pieterse the journal had looked into the situation but found nothing improper. According to an email shared by Pieterse, Nelson wrote:

Because there was no reason to retract the article due to misrepresentation of scientific content, we investigated the issues around withdrawal of patient consent. We have been in communication with the [University of Amsterdam Academic Medical Center] Biobank Committee and Medical Ethics Committee and they have confirmed that withdrawal from the study is not relevant to the article and data that have been published already.

Given the serious implications of a retraction on the journal, the authors of the article, and the scientific record, we have therefore decided that the American Journal of Human Genetics will not retract the article.

In an email to Retraction Watch, Nelson expanded on what he told Pieterse:

Our understanding from the authors and their institutions who obtained and approved consent for this study is that it is possible for research subjects to withdraw their consent at any time and that samples and information should be destroyed upon withdrawal. However, published scientific articles deriving from the studies are not subject to the consent withdrawal and this was confirmed by individuals familiar with European Union Regulations relating to personal data.

Pieterse told us that knows a retraction would be counterproductive to his long-term goal, which is to see the research around Vincents mutation grow. But he still wants to see the OMIM entry come down:

At a certain moment, people are going to cite OMIM in genetics papers and its going to spread. If you want to correct something, you should correct it fast. Once the internet is soaked, you cannot do that.

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Fearing stigmatization, patient's father seeks retraction of paper on rare genetic mutation - Retraction Watch (blog)

Dr. Shigeaki Hinohara died last month at age 105. Over his long life, he helped many others achieve long lives by popularizing annual medical checkups and by sharing what he knew, which was one of his tips for longer living:

He also wrote a musical for children when he was 88 and a best-selling book when he was 101. He recently took up golf. Until a few months ago he was still treating patients and kept a date book with space for five more years of appointments.

Here's the short and sweet version based on this list:

He also suggested finding a job where you never want to retire, and taking the time to enjoy music and animals.

10 Longevity Tips from Dr. Shigeaki Hinohara, Japans 105-Year-Old Longevity Expert (Open Culture via NYT)

Oriental Rat Flea (Xenopsylla cheopis) SIZE: Up to 1/6 in (4 mm) FAMILY: Pulicidae HABITAT: Near rats, their primary food source DISTRIBUTION: Worldwide, particularly tropical and subtropical climates, but some temperate zones as well MEET THE RELATIVES: The cat flea, Ctenocephalides felis, is a relative, as is the dog flea C. can isbut in the []

The establishment wing of the Democratic Party has spent decades receiving dump-trucks full of money from the insurance industry and then, totally coincidentally, explaining that the time isnt right for single-payer for completely unrelated reasons.

Once big data systems agglomerate enough data about you to predict whether you are likely to get sick or badly injured, insurers will be able to deny coverage (or charge so much for it that it amounts to the same thing) to anyone who is likely to get sick, forcing everyone who might ever need []

You dont always have to pay out the nose for household items, everyday accessories, or memorable gifts. If youre searching for something unexpected that can be had for less than two sawbucks, take a look at the following goods:20oz Insulated Water Bottle ($18.99)This stainless steel water bottle is double-walled with vacuum-sealed insulation to keep drinks []

For anyone thats always travelling between their home, office, storage unit, art studio, or any other location thats behind a locked door, you probably have more keys than you know what to do with. Fortunately, the Extended KeySmart keeps them all in a single, compact place that mitigates the need for a huge janitor key []

Working remotely often means using a full-size laptop, or forcing a tablet to do things it was never intended to do. Depending on your job, each may be a reasonable, if somewhat compromising solution, or an impossibly frustrating one. Either way, youll be stuck with a tiny screen and a form factor that will destroy []

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Lifehacks from a longevity expert who just died at age 105 / Boing ... - Boing Boing

New study finds that the exceptionally long lifespan of the Greenland shark may provide clues to extending human lifespan.

About Greenland Sharks

Greenland sharks are considered the longest living vertebrates. A team of scientists led by Julius Nielson, a marine biologist from the University of Copenhagen, used radiocarbon dating on 28 Greenland sharks. They established that one of the female sharks was approximately 392 years.

Research Surrounding the Greenland Sharks

The longevity of the Greenland sharks has stirred interest in the research world. Scientists have worked towards identifying the genes responsible for this unique trait.

Could these genes be used to extend the lifespan of human beings? Scientists believe that a successful identification and extraction of the genes could be a major milestone in achieving longevity among human beings.

Researchers at the University of Exeter have studied almost 100 Greenland sharks. They have sequenced their mitochondrial genome. The achievement set an excellent stage for researchers to scrutinize the sequence and understand the reason behind the shark's longevity.

Prof Kim Praebel, the lead scientist of the research, explained why the research is vital. He noted that unraveling the secrets behind the Green shark's longevity may tremendously boost the efforts on the improvement of humans' lifespan.

Why is the Study of Greenland Sharks Important?

Discovery of the genes responsible for longevity in Greenland sharks would be a vital achievement in the research world. It would explain the limited life spans in almost all the vertebrates. Additionally, the discovery would allow the scientists to study whether the genes can be used to prolong the life of the human beings.

The research at the University of Exeter found out that the Greenland sharks swim long distances across the Atlantic Ocean. They discovered that they mate in the deeply hidden fjords of the Arctic. This realization is instrumental in uncovering the negative effects of the activities of human beings on aquatic life. The study of the tissues and the bones of the animal forms genetic data that is crucial in determining when contaminants and chemicals from industries started affecting the marine. The findings would be of considerable benefit to the environment and marine life protection.

The findings of this research have been presented in various forums including the Fisheries Society of the British Isles symposium. The scientists hope that the findings will boost the conservation efforts of the Greenland shark species and other wildlife in the ecosystem.

Read the rest here:
Do Sharks Hold the Secret to Longevity? - Anti Aging News

The police departments number one priorityreducing gun violenceis being achieved and the crime stats prove it: Five homicides compared to seven last year, and a shining achievement compared to the states comparable big cities, where to date Hartford has 19 homicides and Bridgeport 18.

But would a diminishing of resources due to the ongoing state budget crisis put the brakes on that positive momentum?

No, and, yes. Possibly.

Those achievements, combined with a touch of low-grade anxiety about the resources to continue to advance them, emerged in a candid discussion at Tuesday nights regular meeting of the Board of Police Commissioners.

Commissioner Donald Walker asked about the departments plans for the continuing [state] fiscal crisis.

Mayor Harp is committed to not cutting public safety, Chief Anthony Campbell replied. He said the department is not anticipating the need to cut any officers.

However, if things dont change soon, I can see our going to zero overtime. Were not there yet and we hope and pray we dont, and we have a plan [in place] for that, he added.

That zero overtime plan would affect the configuration of the shifts and other deployments, but it should not affect public safety.

One area, where the states fiscal woes are already being felt: Project Longevity.

That project launched here in November 2012 by the city, the U.S. attorney, and the governor, identifies the small number of gang-related young men most involved in violence. It brings them to carefully choreographed call-ins to hear from law enforcement officials and community leaders. They hear a plea to stop the violence. Then they get a choice: Take advantage of immediate help in finding jobs, housing, medical care or earning degrees to straighten out their lives. Or go back to shooting in which case local, state and federal agents will come down on their entire groups to put them behind bars for decades under federal mandatory minimum sentencing laws. (Click here for a story on how the project helped fell one deadly gang.)

New Havens project has been successful. (An example was announced on Wednesday, with the arrests of six alleged New Haven gang members, affiliated with the Goodrich Street Boys gang, on federal attempted murder, firearms, narcotics, and racketeering charges.)

Assistant Chief Archie Generoso, who also presented at the commissioners meeting, attributed much of the reduction in gun violence to the project: Between 2003 and 2012 there were 126 shootings. However, since the advent of Project Longevity in November 2012, only 64 shootings have been logged in. We cut it in half, said Generoso.

That was one reason why already eight cities have come to study New Havens approach, including, Generoso added, officials from Honduras arriving in town next week for that purpose.

The anxiety centered on the paycheck of the exceptional former police officer who runs Project Longevity, Stacy Spell. Only he and an assistant are paid all the other officers who participate do it on their own time and they are paid by the cash-strapped and budget-stalled state. The state faces a $5-plus billion two-year deficit and is already more than a month late in trying to pass a new budget.

He hasnt received a pay check in a month, due to the state budget stalemate, Generoso reported.

Commissioner Stephen Garcia asked how the department is supporting Spell.

Stacy is doing an unbelievable job, said Genoroso.

Despite not being paid, Chief Campbell added.

Commission Chair Anthony Dawson said he has some ideas to address the situation, which will be discussed at another time.

The chiefs also reported that 29 new officers recently completed their field training and are ready to be deployed. They will reduce our overtime by $15,000 a day, said Campbell.

After presenting the crime stats, Generoso concluded: Well not be satisfied until zero [homicides]. We are bucking national trends. Our numbers are going down, while nationally the numbers are going up. Were making it happen. Its due to the support from you, the alders, the mayor. They have not cut back on money and resources. This city invests in the safety of its citizens.

Following the tragic shooting of 14-year-old Tyriek Keyes on July 16, not only are more police patrolling Newhallville. There are more crews of tree-trimmers at work there as well.

That news emerged in a report by Assistant Chief Generoso at Tuesday nights commissioners meeting.

For the last eight days two of the Department of Parks, Recreation and Trees four contract tree-trimming crews have been assigned to the streets around the Lincoln-Bassett School, the neighborhood where Tyriek was killed.

While tree-trimming is part of an ongoing citywide program, the Department Director Rebecca Bombero said that for the first time we did a ride-along with the police to scope some of their requested safety trim, which resulted in a larger scope of work. The focus was in Newhallville around Lincoln Bassett.

That scope includes raising trees to improve site lines for cameras, traffic signals, and lighting, she added.

At Tuesday nights meeting, Assistant Chief Generoso reported to the commissioners that the stepped up police patrolling was being accompanied by this important arboreal work, with an aim to cut some of the trees around some of our cameras and lights.

The city is in the process of putting up more cameras in Newhallville and elsewhere, he added. Cutting trees that obscure lights and camera views is work Generoso described as essential to enhance safety and crime-solving.

He said that officers indicated areas where the branch trimming is a priority and that the parks and rec crews should be finishing up in Newhallville this week.

Bombero wrote in an email that her department has over 2,000 open issues, or trimming requests, at any given time. The staff to respond to all that consists of two internal crews, along with contract crews funded through a capital allocation centered around hurricane season.

This year the funding has allowed for four crew, with the priorities being immediate hazards, safety trimming as requested by police, engineering or [the department of] Transportation, Traffic & Parking for site lines and secruity and then by level of hazard by date reported, she wrote.

Call your state legislators. They are doing nothing but waiting to be told how to vote. They demand more and higher taxes. They dont cut costs. They are bankrupt of ideas and they are hiding from this entire budget issue. This issue has been around since fxxking February - It is not August. The budget is late, the excuses are lame and plentiful. Meanwhile, they all pose for selfies, go live and one even went to Boston for a conference on Being Ready from the Inside. lol - you just cant make this stuff up. Meanwhile, this program suffers; schools are laying off teachers and others; not one community can do anything. Even well run East Haven has a hiring and spending freeze on except for the basic stuff.

The stupidity of not having a budget this late in the yar; of operating on a day to day basis by executive order, causing non-profits to close, of homeless shelters for women and kids to close - these nitwits couldnt even pass a mini-budget while they traveled and played golf. Oh, and collected their nice paychecks, perks and accumulated their retirement benefits. Amazing.

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State Budget Woes Threaten Project Longevity - New Haven Independent

First-half operating profits at Legal & General climbed 27% year-on-year to 988m ($1.28bn), with more than half of the increase arising from a review of longevity assumptions in its annuity book.

Decades of sustained improvement in longevity in the UK appears to have reversed in recent years, in particular among older age groups. Commentators have attributed this to a variety of causes, such as less government spending on health and old-age care, and virulent winter flu outbreaks.

The trend towards lower longevity improvements means that annuity underwriters may have to set aside fewer reserves, and has led some analysts to consider how much reserve could be released and returned to shareholders.

In todays half-year results announcement, L&G revealed it had released 126m of reserves. But this was based on a review of its base mortality assumptions the current levels of mortality being experienced by pensioners not the mortality trends.

In preparing the half-year results, we have not adjusted our assumptions for the rate of future longevity improvement; they remain consistent with those disclosed last year.

L&G said it will review its longevity improvement assumptions at the year end, including the appropriateness of using theContinuous Mortality Investigation (CMI) 2015 model.

There is increasing evidence that the higher than expected level of recent mortality is in part due to medium or long-term influences rather than short-term events. In performing this review, consideration will be given as to whether, and over what period, to move to newer versions of the CMI model.

L&Gs Retirement division responsible for writing individual and bulk annuities, longevity insurance and equity-release mortgages has a gross longevity exposure of 61.4bn across its annuity and longevity insurance business. The firm has reinsured 15.9bn of longevity risk with 11 reinsurance counterparties, leaving a net exposure of 45.5bn. Going forward, the firm plans to reinsure 80-90% of longevity risk from new bulk annuity business.

L&Gs Solvency II ratio climbed by 15 percentage points over the six months to 30 June, to reach 180%. The figure incorporates an estimated impact from recalculating the transitional measures for technical provisions (TMTP) as at 30 June.

Christopher Cundy

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Longevity review boosts L&G's half year profits - insuranceERM

A shot of klotho, a hormone associated with longevity, seems to make mice smarter. Klotho is a naturally occurring hormone in the body. More than two decades ago, Japanese researchers discovered that this hormone plays a role in aging. People with more klotho in their body, tend to live longer and to retain more of their facultiesthat is to stay sharpwell into old age.

Researchers injected three types of mice with a portion of the protein. They injected young mice, aged mice, and mice genetically altered to have brains similar to that which we would see in Alzheimers or Parkinsons patients in humans.

Within hours they showed better cognitive function, says Dubal.

Since you cant exactly administer a mouse an IQ test, they assessed brain power based on the mices ability to navigate a series of water mazes, in an experiment that sounds on par with human a trip to Wisconsins famed waterslide park, The Dells.

They found that mice that had daily injections and were better able to navigate the maze (as measured by the distance traveled to find a hidden platform) than their control group peers. In a classic example of work smarter, not harder, the klotho mice were just much more efficient seekers.

We tested them two weeks later in a different cognitive test and they were still smarter, says Dubal, which suggested that getting the klotho protein into their bodies combined with brain training and stimulation had a long-lasting effect in their brain. Because the half-life of the protein is only seven and a half hours long, any of the protein should have been long out of their system.

Cell Reports Peripheral Elevation of a Klotho Fragment Enhances Brain Function and Resilience in Young, Aging, and -Synuclein Transgenic Mice

Highlights

A klotho fragment (KL-F) enhances cognition in young and aging mice KL-F counters deficits in -synuclein mice without altering pathogenic protein levels KL-F induces GluN2B cleavage and increases NMDAR-dependent synaptic plasticity Selective NMDAR blockade of GluN2B subunits abolishes acute KL-F effects

Summary

Cognitive dysfunction and decreased mobility from aging and neurodegenerative conditions, such as Parkinson and Alzheimer diseases, are major biomedical challenges in need of more effective therapies. Increasing brain resilience may represent a new treatment strategy. Klotho, a longevity factor, enhances cognition when genetically and broadly overexpressed in its full, wild-type form over the mouse lifespan. Whether acute klotho treatment can rapidly enhance cognitive and motor functions or induce resilience is a gap in our knowledge of its therapeutic potential. Here, we show that an -klotho protein fragment (KL-F), administered peripherally, surprisingly induced cognitive enhancement and neural resilience despite impermeability to the blood-brain barrier in young, aging, and transgenic -synuclein mice. KL-F treatment induced cleavage of the NMDAR subunit GluN2B and also enhanced NMDAR-dependent synaptic plasticity. GluN2B blockade abolished KL-F-mediated effects. Peripheral KL-F treatment is sufficient to induce neural enhancement and resilience in mice and may prove therapeutic in humans.

Introduction

Cognitive dysfunction and decreased mobility from aging and age-related neurodegenerative conditions such as Alzheimer disease (AD) and Parkinson disease (PD) are major biomedical challenges. Because more effective treatments are needed, and clinical trials targeting putative pathogenic proteins have failed, it is critical to develop alternate or complimentary therapeutic strategies. In light of this urgent medical need for our rapidly aging populations, delaying aging itself or increasing the function and resilience of the brain (Bennett, 2017, McEwen and Morrison, 2013) may represent new treatment strategies.

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Klotho longevity hormone helped make mice smarter - Next Big Future

Wayne Gretzky knows a thing or two about being the best in his discipline, and it comes as no surprise to the hockey legend that Rafael Nadal and Roger Federer are currently battling for No. 1 in the Emirates ATP Rankings, more than a decade after the pair first dominated the ATP World Tour.

Oh yeah, when youre that good and that dedicated, anything is possible, Gretzky exclusively told ATPWorldTour.com in Montreal on the opening day of main draw action at the Coupe Rogers.

Gretzky hung up his skates at the age of 38, finishing his career with four Stanley Cups, an all-time best 894 goals and countless records in 20 NHL seasons. Federer, a five-time year-end World No. 1, celebrates his 36th birthday on Tuesday. The 31-year-old Nadal finished World No. 1 in 2008, 10 and 13 and this week has a chance to return to the top spot for the first time since July 2014.

Together this season, the rivals and friends have swept three Grand Slam titles and four of the five ATP World Tour Masters 1000 tournaments. They have faced off three times, including a memorable five-set win for Federer at the Australian Open.

As a sports fan, I admire when a player can be at an elite level, said Gretzky. To get to an elite level is really hard, but to stay there for a long period of time obviously Roger has been there a little bit longer, is a little bit older than Nadal but it really is incredible for sports and its really fascinating to see two players of that caliber compete as hard as they do against each other, yet have so much respect for each other.

If you have a passion for what youre doing, you go to another level, he added. I think the thing that separates stars from superstars is that superstars want to be under pressure. They want to be under the microscope. They want to play in the finals. They want to be 2-2 going into the fifth set. Thats where theyre comfortable. You see players like Nadal and players like Djokovic and obviously Federer, and going back to guys like Bjorn and McEnroe, they want to be under the gun. It makes them play that much better, the bigger the game the more relaxed they seem to be and the more inspired they are and the better they play.

On Monday, The Great One strolled around the Stade Uniprix grounds, making his rounds of the practice courts to show his support for the home favourites. After greeting top Canadian Milos Raonic, Gretzky headed over to Court 8 to watch up-and-coming Canadian Denis Shapovalov during a practice session with another #NextGenATP star, Korean Hyeon Chung. Gretzkys 14-year-old daughter Emma, who has been playing tennis for a year, also had an opportunity to hit with the Bryans as they wrapped up practice on the adjacent court.

Thats the great thing with sports, theres always the next person that comes along Guys like Milos and Daniel Nestor have opened doors for the young guys to come along, and years from now hopefully theyll open doors for the new generation of kids that come along 10-15 years from now, said Gretzky.

The Brantford, Ontario native, who also frequents the BNP Paribas Open in Indian Wells, said he enjoyed the level of competition at the ATP World Tour Masters 1000 tournaments and planned to be back on site throughout the week to watch some matches.

Its always fun to have a big event like this in Canada, he said. Everyone enjoys seeing the best players compete against each other. In our country, we rally around supporting our local favourites, whether its Milos, Denis this year now, Genie Bouchard in Toronto. Were very proud of our kids who compete against the best in the world and thats what makes it fun for Canadians to watch.

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Gretzky Calls Federer & Nadal's Longevity On Top 'Incredible' - ATP World Tour

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A lab technician at Myriad Genetics in Salt Lake City, Utah.

Thousands of people are getting genetic tests, for everything from their cancer risk to their likelihood of passing on a disease to a child.

But many doctors aren't adequately trained to interpret these results, or tell patients how to act on them. And genetic counselors -- who do have that knowledge -- are in short supply. There are only about 4,000 genetic counselors in the country today. That's one for every 80,000 Americans. That means some patients have to wait months to get a consultation.

Start-up Clear Genetics, which launches this week after raising $2.5 million in financing, is trying to make genetic expertise more widely available.

The start-up has developed a conversational chatbot to guide a user through their results, collect information and review options for genetic testing, and answer questions about things like whether the test will be covered by insurance. If there's a need for additional support, the patient can then schedule a consultation with a human expert via video or in-person.

"We're finding that it's working really well with patients," said Moran Snir, Clear Genetics' CEO, who was previously a software engineer with the Israeli military.

Clear Genetics is working with several large health systems in the United States to test out a beta version of its product.

"I think this is a very good use for AI," said David Ledbetter, executive vice president and chief scientific officer at hospital network Geisinger Health System, in an interview with CNBC.

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You're getting a DNA test start-up Clear Genetics is building chatbots to help you understand the results - CNBC

Genes carry the information that make you you. So it's fitting that, when sequenced and stored in a computer, your genome takes up gobs of memoryup to 150 gigabytes. Multiply that across all the people who have gotten sequenced, and you're looking at some serious storage issues. If that's not enough, mining those genomes for useful insight means comparing them all to each other, to medical histories, and to the millions of scientific papers about genetics.

Sorting all that out is a perfect task for artificial intelligence. And plenty of AI startups have bent their efforts in that direction. On August 3, sequencing company Veritas Genetics bought one of the most influential: seven-year old Curoverse. Veritas thinks AI will help interpret the genetic risk of certain diseases and scour the ever-growing databases of genomic, medical, and scientific research. In a step forward, the company also hopes to use things like natural language processing and deep learning to help customers query their genetic data on demand.

It's not totally surprising that Veritas bought up Curoverse. Both companies spun out of George Church's prolific Harvard lab. Several years ago, Church started something called the Personal Genomics Project, with the goal of sequencing 100,000 human genomesand linking each one to participants' health information. Veritas' founders helped lead the sequencing partstarting as a prenatal testing service and launching a $1,000 full genome product in 2015while Curoverse worked on academic strategies to store and sort through all the data.

But more broadly, genomics and AI practically call out for one another. As a raw data format, a single person's genome takes up about 150 gigabytes. How!?! OK so, yes, storing a single base pair only takes up around two bits. Multiply that by roughly 3 billionthe total number of base pairs in your 23 chromosome pairsand you wind up with around 750 megabytes. But genetic sequencing isn't perfect. Mirza Cifric, Veritas Genetics cofounder and CEO, says his company reads each part of the genome at least 30 times in order to make sure their results are statistically significant. "And you gotta keep all that data, so you can refer back to it over time," says Cifric.

That's just storage. "Everything after that is going to specific areas and asking questions: Theres a variant at this location, a substitution of this base, a deletion here, or multiple copies of this same gene here, here, and here," says Cifric. Now, interpret all that. Oh, and do it across a thousand, hundred thousand, or million genomes. Querying all those genetic variations is how scientists get leads to find new drugs, or figure out how existing drugs work differently on different people.

But cross-referencing all those genomes is just the beginning. Curoverse, which was focusing on projects to store and sort genomic data, also has its work cut out for it in searching through the 6 millionand countingjargon-filled academic papers detailing gene behavior, including visual information found in charts, graphs, and illustrations.

That's pretty ambitious. Natural language processing is one of the stickiest problems in AI. "Look, I am a computer scientist, I love AI and machine learning, and no amount of coding makes sense to solve this," says Atul Butte, the director of UCSF's Institute of Computational Health Sciences. At his former job at Stanford University, Butte actually tried to do the same thinguse AI to dig through genetics research. He says in the end, it was way cheaper to hire people to read the papers and input the findings into his database manually.

But hey, never say never, right? However they accomplish it, Veritas wants to move past what companies like 23andMe and Color offer: genetic risk based on single-variant diseases. Some of America's biggest dangers come from diseases like diabetes and heart disease, which are activated by interactions between multiple genesin addition to environmental factors like diet and exercise. With AI, Cifric believes Veritas will be able to not only dig up these various genetic contributors, but also assign each a statistical score showing how much it contributes to the overall risk.

Again, Butte hates to be a spoilsport, but ... there's all sorts of problems with doing predictive diagnostics with genetic data. He points to a 2013 study that used polygenic testing to predict heart disease using the Framingham Heart Study dataabout as good as you can get, when it comes to health data and heart disease. "They authors showed that yes, given polygenic risk score, and blood levels, and lipid levels, and family history, you can predict within 10 years if someone will develop heart disease," says Butte. "But doctors could do the same thing without using the genome!"

He says the problems come down to just how messy it is trying to square up all the different research on each gene alongside the environmental risks, and all the other compounding factors that come up when you try to peer into the future. "Its been the holy grail for a long time, structured genome reporting," says Butte. Even attempts to get researchers to write and report data in a standard, machine-readable way, have fallen flat. "You get into questions that never go away. One researcher defines autism different from another one, or high blood pressure, or any number of things," he says.

Butte isn't a total naysayer. He says partnerships like the one between Veritas and Curoverse are becoming more commonlike the data processing deal between genetic sequencing giant Illumina and IBM Watsonbecause there's a clear need for new computing methods in this area. "You want to get to a point where you are developing stuff that improves clinical care," he says.

Or how about directly to the owners of the genomes? Cifric hopes the merger will improve the consumer experience of using genetic data, even seamlessly integrating it into daily life. For instance, linking your genome and health records to your digital assistant. Alexa, should I eat this last piece of pizza? Maybe you should skip it, depending on your baseline genetic risk for cholesterol and latest blood test results. Diet isn't the only area where genomics could help improve your day to day life. Some people are more or less sensitive to over the counter drugs. A quick query might tell you whether you should take a little less Tylenol than is recommended.

Cifric thinks this acquisition could position Veritas as a global powerhouse of genomic data. "Apple recently announced that they had shipped 41 million iPhones in a quarter, right? I think in not too distant future, well be doing 41 million genomes in a quarter," he says. That might seem ambitious, given that the cost to consumers is nearly $1,000. But that cost is bound to come down. And artificial intelligence will make paying for the genome a matter of common sense.

This story has been updated to reflect that the company is named Veritas Genetics, not Veritas Genomics.

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Veritas Genetics Scoops Up an AI Company to Sort Out Its DNA - WIRED

DURHAM Millions of people die every year from dehydration as a result of exposure and illness. In humans, even the most minor dehydration can compromise the kidneys causing lifelong, irreparable issues or even death. However, some animals living in desert environments are able to survive both acute and chronic dehydration. While these animals, like cactus mice, have evolved over time to deal with environmental stressors like dehydration, researchers at the University of New Hampshire have found its not the physical makeup that is helping them survive, but rather their genetic makeup.

Initially, we thought that maybe their kidneys are structurally different from people, but theyre not, said Matt MacManes, assistant professor of genome enabled biology at UNH and lead author of the study. However, when exposed to acute dehydration, no kidney injury was apparent, which would definitely be the case for humans exposed to similar levels of dehydration, suggesting their genes may be whats preventing widespread kidney damage.

The kidney is the canary in the coal mine when it comes to dehydration, continues MacManes. The exciting outcome of this research is that the molecular toolkit of the cactus mouse has orthologues, or related genes, in humans. These provide the potential for development of drugs or other therapies that could help protect the human body from the damages of dehydration. Such a response could be extremely valuable in a wide variety of situations for people with renal failure, where water is severally limited due to geography or possibly global climate change, for troops deployed in the desert, and perhaps even in space travel.

To understand how desert-adapted cactus mice (Peromyscus eremicus) survive, the study recently published in the American Journal of Renal Physiology outlines how the researchers modeled a desert-like condition. The mice that went without water for 72 hours lost on average 23 percent of their body weight, which would be fatal for humans. Even though dehydrated, the mice continued to be active, eat, and interact normally. Researchers analyzed several other factors including serum electrolytes (sodium, calcium, bicarbonate ion) as well as blood urea nitrogen (BUN) and creatinine. While both were slightly elevated, gene-based biomarkers for kidney injury, were not, which suggests kidney injury is not occurring.

Further analysis found genes that are important in modulating electrolytes were very active, as were genes responsible for maintaining kidney blood pressure.

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UNH research: Genetics mechanism preventing kidney injury after severe dehydration - Foster's Daily Democrat

It appears, by all accounts, to be a momentous scientific achievement and possibly a turning point in human evolution. In a study released last week, scientists at Oregon Health and Science University confirmed they were able to modify genes in viable human embryos, proving the potential to permanently alter the makeup of a genetic line.

In this case, that meant replacing and repairing a mutated gene that causes a common and deadly heart disorder. But the possibilities heralded by gene-editing technology are endless, the scenarios as divided as they are bold. In some visions, it leads to a population of designer babies or consumer eugenics. Others imagine a utopia of scientific advancement where humans live free of disease, and devastating conditions are eradicated for the betterment of humanity. What direction the technology will take is the topic of much debate.

The big thing which is making the scientific and ethics community get excited, and on the other hand a little bit hot and bothered, is its a mechanism to change genes for multiple generations, says Dr. Alice Virani, a genetic counsellor and director of ethics at British Columbias Provincial Health Services Authority. There are two ways to look at it, the more realistic ramifications and the sci-fi, if-this-was-out-of-control ramifications.

Opinion: Gene editing is not about designer babies

The team at the Oregon universitys Center for Embryonic Cell and Gene Therapy used technology called CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, to repair or edit the gene carrying the heart disorder, seemingly with greater success than previous attempts by scientists in China.

News of the research has been anxiously anticipated by many in the field, both for what it means for the potential eradication of a disease such as hypertrophic cardiomyopathy and for the fundamental questions it raises about human reproduction, health and society.

When the study was leaked days before its publication in the journal Nature, its lead scientist, Dr. Shoukhrat Mitalipov, attributed the release to likely a combination of hot words: CRISPR, gene-editing, and designer babies.

The study and its combination of hot words didnt disappoint.

The New York Times hailed the milestone in research, while The New York Post cried BABE NEW WORLD and described an amazing and slightly terrifying breakthrough. A headline on Vox declared simply, This Is Huge.

Even actor Ashton Kutcher tweeted enthusiastically about the scientific breakthrough, writing: Scientists successfully used CRISPR to fix a mutation that causes disease. This is why I wanted to be a geneticist!

The tweet ignited among his followers the same range of responses that are always so keenly tied to the issue of changing human genes, from hope that devastating conditions such as muscular dystrophy will be eradicated, to fear about the unknown consequences of playing God.

Dr. Timothy Caulfield, a Canada Research Chair in Health Law and Policy and professor at the University of Alberta, says the polarized and dramatic response he has seen in recent days reminds him of early reaction to stem-cell science, where, he says, It was either going to be cloned armies, or we were going to eradicate all disease.

In fact, neither has turned out to be the case, and so it may be with gene editing as well.

We need to be cautious not to hype the benefits and be cautious not to hype the ethical concerns, he says. There are real issues on both sides of the debate but lets make sure our discourse is evidence-formed.

He described the new research as a genuinely exciting area, and said the potential of CRISPR which is used not only in human genetics, but also has potentially revolutionary applications for agriculture, animals, plants and food has introduced both exciting possibilities and reasons for deep policy reflection.

Erika Kleiderman, a lawyer and academic whose work focuses on gene-editing technologies, stem-cell research and regenerative medicine at the Centre of Genomics and Policy at McGill University, says the Oregon teams research is exciting because it confirms the ability of CRISPR technology to repair genetic mutations, and establishes the basic safety of the technique in a research context. And while she said people often go straight to thinking about the potential for manipulating genes to create so-called designer babies, a concept that is cool but also quite frightening, the medical implications could be equally staggering, and are far more likely.

For example, something like Huntington disease, she says. Being able to prevent that or treat that one day, in my opinion, would be a fantastic leap for our scientific knowledge and medical advancement. That being said, people will raise the eugenics argument. Is that a possibility? Yes. Are we close to that? I dont think so.

Canada has strict laws around genetic modification and editing, and altering genes in a way that could be passed on to future generations is a criminal offence under the Assisted Human Reproduction Act, punishable with fines up to $500,000 or 10 years in prison.

But as the technology takes a large step forward, Ms. Kleiderman and Dr. Caulfield and are among a group of Canadian scientists and academics calling for less regulation around genetic science and research in Canada, not more.

Both were involved in the creation of an editorial published in the journal Regenerative Medicine in January calling for new consideration of the issues and ethics involved in gene editing, and a revision of Canadian legal policy.

A criminal ban is a suboptimal policy tool for science as it is inflexible, stifles public debate, and hinders responsiveness to the evolving nature of science and societal attitudes, the editorial read. It was signed by seven other experts and ethicists, and came out of a think tank on the future of human gene editing in Canada held at McGill last summer.

Dr. Caulfield says legal prohibition of certain genetic research doesnt make sense when we dont yet know or understand where the science is going, or what the benefits or harms could be. Instead, he says he believes in regulation in problematic areas, while allowing for studies and trials. He says that some of the slippery slope scenarios people fear such as using genetic modification for human enhancement and to achieve superficial traits such as height remain distant possibilities given the complexity of the science.

That is not to say there are not risks or issues to be addressed as the technology continues to evolve. Ms. Kleiderman says that includes consideration of the potential risk to future generations, the safety of the technology and other irrevocable, if unintended, consequences, although she says those risks are not unique to gene modification but true of all technologies.

When it comes to CRISPR, one of the areas it would be most beneficial is with the treatment of prevention of disease which I think most people would be in agreement with, she says. Of course, we need to be mindful of doing not-so-positive things with it, like going down the enhancement route.

She said other potential issues, such as the preservation of human diversity and individuality, the welfare of children born from this technology and the potential for creating new forms of inequality, discrimination or societal conflict, all require significant consideration and research.

There is time. Although the technology is moving quickly, there is still a long way before gene editing is used in clinical human trials. Even after that, Dr. Virani says for the foreseeable future the technology will most likely be used by a small group of people in specific scenarios related to the prevention of serious genetic disease.

Im not saying we shouldnt be concerned about those potential issues, but sometimes we make that leap too quickly, she said. We dont necessarily [think] that the most likely scenario is that couples will use this technology on a very limited basis if they know their child may potentially have a devastating genetic condition. Thats not something that suddenly everyone is going to start to do. I think theres sometimes that leap to, Oh, we can create designer babies, but I think were very much in the lessening-burden-of-disease phase rather than the designer-baby phase, though thats where peoples minds go.

Dr. Virani said one of her own concerns is the possibility of off-target effects, where changing a gene unexpectedly alters something else in the genome. Other concerns are more social reality than science fiction, including that the technology and the ability to prevent disease may only be available to those who can pay for it. Eradicating a horrible disease is one thing. Eradicating it only for families who can afford it is another.

So is it going to look like just the wealthy are going to be able to afford this type of technology? she asks. Thats very problematic in my eyes from an ethics point of view, and thinking about fairness in society. If only poor people get Huntington disease, then the lobby to support Huntington disease research is greatly diminished. Its kind of like a two-fold negative effect.

On Thursday, the American Journal of Human Genetics ran a policy statement signed by 11 organizations from around the world, including the Canadian Association of Genetic Counsellors, urging a cautious but pro-active approach as the science moves forward. The statement includes an agreement that gene editing should not yet be performed in embryos carried on to human pregnancy. (The embryos used in the Oregon research were created only for the research, and were not developed further.) It also outlines a number of criteria that should be met before clinical trials take place, and supports public funding for the research. The U.S. government does not allow federal funding for genetic research on embryos. The Oregon research was funded by the university.

We dont want it to go speeding ahead, said Kelly Ormond, the lead author of the policy statement and a genetics professor at Stanford University in California. We want people to be very transparent about whats happening and we want things to undergo good ethics review, and for society to actually be engaged in these dialogues now while this research is just starting to happen.

She said she believes its important to be pro-active in talking and thinking about the issues related to the technology, and starting a broader conversation of how gene editing should and will be used.

We can all agree that that world [of eugenics and designer babies] doesnt feel very comfortable, and I think most of us dont want to go there, she said. So we need to find ways to prevent that from happening.

Follow Jana G. Pruden on Twitter: @jana_pruden

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Modification of genes in human embryos could mark turning point in human evolution - The Globe and Mail

The global market for stem cells has been estimated at USD 12 billion in 2016and is projected to reach USD 26.6 billion by 2021, at a CAGR of 13.7% during the forecast period 2016to 2021. A stem cell is an undifferentiated cell that has the potential to develop into any type of cell in the body.

Regenerative medicine is the major application of stem cells and other areas are neurology, orthopedics, oncology, cardiology, hematology and others (diabetes, injuries, and wounds). Another prominent application of stem cells is drug discovery and development. The end-users of this market are usually hospitals, cell banks, clinical research laboratories and academic institutes.

Global Stem Cell Marketing Market Dynamics

The global stem cells market is one of the most promising markets in the field of life sciences at present and is forecasted to grow even more in the coming years as stem cells enable cost-effective treatment of many conditions that currently have poor or no treatment.

Drivers

Some of the factors driving the global stem cells market are:

Restraints

While the global stem cells market has ample scope for growth, there are some factors restraining it as well. These include:

The market for stem cells is segmented on the basis of cell types and technology. The cells type segment includes adult stem cells, human embryonic stem cells, induced pluripotent stem cells, rat neural stem cells and very small embryonic-like stem cells. Adult stem cells are again divided into hematopoietic stem cells, mesenchymal stem cells, neuronal stem cells, dental stem cells and umbilical cord cells. The adult stem cells hold the highest share in the global stem cells market, while the market share of induced pluripotent stem cells is expected to grow in the coming years. The technology segment is divided into stem cell acquisition, stem cell production, stem cell cryopreservation, and stem cell expansion sub-segments.

Based on geography, the global market for stem cells is segmented into North America, Europe, Asia-Pacific and Rest of the World. The global stem cells market is dominated byNorth America, followed byEurope, the estimated market share of which is more than 25% as per a recent study. With 30% of the market, the USA holds the majority of share. However, due to increasing awareness among the public and advances in technologies, the market in the Asia-Pacific is expected to grow at a high rate.

Many players in this market are trying to expand their product portfolio in order to top the global market. While some companies are entering into the market by acquisitions, top companies are expanding their growth in this market by acquiring other companies. Few companies have adopted product innovation and new product launches as their key business strategy to ensure their dominance in this market.

Some of the key players in the market are:

Key Deliverables in the Study

If you are in need of BioTechnology marketing or Stem Cell Marketing call 972-800-6670

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BioTech Marketing and market opportunity for Stem Cells - Checkbiotech.org (press release)

Stem cells are the highly versatile spare tires of your body. Once called on, they can replace a damaged cell and, because they aren't yet directed to become part of a specific organ or tissue type, they not only could become (metaphorically speaking) a new tire, but could also fix a worn-out engine part or a cracked windshield. It just takes the right prodding in the body, or the laboratory! They can do it even after being inactive for a long time.

Those remarkable abilities are promising to provide scientists with a powerful tool to use in conquering disease. That's because normally, cells in organs such as the heart and pancreas do not divide to repair damage that might happen to the organ. But manipulation of stem cells ... well, that could allow doctors to induce self-repair in many parts of the body. No more heart transplants; bye, bye diabetes, macular degeneration, spinal cord injury, osteo- and rheumatoid arthritis. We might even repair third-degree burns and stroke damage that was previously considered permanent.

That promising future became more hopeful in 2006, when researchers figured out how to turn specialized adult stem cells (replacing use of embryonic cells in some research) into what they called "induced pluripotent stem cells" (iPSCs). Since then, the number of experiments using iPSCs has sky-rocketed: Adult mouse stem cells are injected into the damaged ventricular wall of a mouse heart and the stem cells regenerated damaged heart muscle! There have been a few, small, human-based studies that, says the National Institutes of Health, have "demonstrated that stem cells that are injected into the circulation or directly into the injured heart tissue appear to improve cardiac function and/or induce the formation of new capillaries." But and this is a big but they caution, "significant technical hurdles remain that will only be overcome through years of intensive research."

Tip: Stem cell clinics promising miracle cures are not a good idea at this time. The International Society for Stem Cell Research says: "Many clinics offering stem cell treatments make claims that are not supported by a current understanding of science."

Fortunately, there's a lot you can do to keep your stem cells healthy and your RealAge younger.

1) Protect your skin from excess sun exposure; use micronized zinc oxide 30 SPF sunscreen. Exposure to ultraviolet radiation from the sun and tanning beds and lamps is a leading cause of melanoma. New research shows that the trigger may be stem cells gone wild; melanoma may be related to the formation of carcinogenic stem cells.

2) Avoid hormone-disrupting chemicals such as BPA in plastics and phthalates in household goods and products. One study found that they disrupt development of stem cells needed for sperm production.

3) Don't overeat; eat whole foods, not chemicals. Steer clear of processed foods that dose you with preservatives, colorings, emulsifiers, added sugars and syrups. Continuous intake of sugary foods reduces stem cell vitality! A lab study found that reducing caloric intake by 20 percent can positively boost stem cell activity. We say, try it five days a month.

4) Get regular exercise. According to a new study out of the University of Rochester, loss of muscle stem cells is the driving force in loss of muscle tone and strength as you age. That makes it increasingly important to get two to three 30-minute sessions of strength-building exercises weekly. Aerobic effort (push it a bit) stimulates some stem cells to produce bone instead of fat.

5) Avoid excess radiation. Exposure to a dental X-ray, PET or CAT scan provides diagnostic benefits without immediate risks. But new evidence shows that accumulative exposure to radiation over a lifetime can have damaging effects on stem cells and organs. Opt for an MRI, not a CAT scan when possible; refuse dental X-rays unless necessary; follow guidelines for mammograms. And make sure your imaging center is accredited, personnel are credentialed, and they use weight-based and indication-based protocols.

Mehmet Oz, M.D. is host of "The Dr. Oz Show," and Mike Roizen, M.D. is Chief Wellness Officer and Chair of Wellness Institute at Cleveland Clinic.

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How to keep your stem cells young - The Garden City Telegram

The advanced technology offered by uBiome may enable us to detect harmful oral bacteria before they endanger the lives of these children - with just a bit saliva.

San Francisco, CA (PRWEB) August 08, 2017

uBiome, the leader in microbial genomics, has issued its latest Microbiome Impact Grant award to pediatric dentist and scientist Dr. Jeremy Horst of UCSF School of Medicine, who, along with colleagues in the UCSF Children's Oral Health Research Center, is carrying out research into the use of the oral microbiome as a non-invasive way of predicting and preventing blood infections in immunocompromised young bone marrow transplant patients.

Bone marrow transplants are used in order to replace damaged or diseased cells with non-cancerous stem cells that can, in turn, grow new, healthy cells. These transplants tend to be used when treatments for cancer have destroyed the bone marrows normal stem cells. Bone marrow, which is found at the core of bones, is where the body manufactures blood cells.

Bone marrow transplants can be either allogeneic or autologous. Allogeneic transplants occur when bone marrow is received from a donor. In autologous transplants, the patients own bone marrow is used, after being collected, frozen, and stored until it is needed following chemotherapy, for example.

Blood infections pose a considerable risk during bone marrow transplants, so being able to predict and prevent them is critical. Dr. Horsts study aims to explore the use of the oral microbiome as a predictive diagnostic for blood infections in pediatric patients who are immunocompromised, a common phenomenon during transplant procedures. Having a weakened immune system, technically known as immunodeficiency, is a state in which the immune systems ability to fight infectious disease and cancer is either compromised or entirely absent.

The potential to use the oral microbiome as a marker for the blood microbiome would offer considerable benefits, particularly because of its non-invasive nature.

Dr. Horst is a Postdoctoral Scholar in the Biochemistry and Biophysics Department at UCSF School of Medicine, specializing in Biochemistry and Infectious Diseases. He received his PhD for studies in Oral and Computational Biology at the University of Washington, after also first gaining his DDS there. This was followed by a residency in Pediatric Dentistry at UCSF. Dr. Horst began his academic studies at UCSD, where he was awarded his BS in Pharmacological Chemistry, a BA in Psychology, and a masters in Chemistry. He has contributed to 40 scientific papers.

The microbiome is the collective term for the ecosystem of trillions of microorganisms that live in and on the human body. Many play important parts in supporting life. For example, gut bacteria aid digestion and enable the synthesis of vitamins. Pathogenic bacteria, however, can be associated with a range of conditions. uBiome employs precision sequencing technology to generate detailed analyses of the human microbiome.

Dr. Jeremy Horst says: To prepare young patients for bone marrow transplant, their immune systems are temporarily wiped out. Despite our extraordinarily cautious efforts, one third of these children at UCSF Benioff Childrens Hospital get blood infections, and oddly enough, one third of the infections come from bacteria in the dental plaque. We use traditional culture-based diagnostics to understand these infections once they happen, but the advanced technology offered by uBiome may enable us to detect harmful oral bacteria before they endanger the lives of these children - with just a bit saliva.

Dr. Zachary Apte, co-founder and CTO of uBiome, adds: After collaborating with him in the past, were familiar with Dr. Horsts work. We think his novel proposal to predict and prevent blood infections in young patients with weakened immune systems using something as simple as a saliva test is very exciting.

Founded in 2012, uBiome is the worlds leading microbial genomics company. uBiome is funded by Y Combinator, Andreessen Horowitz, 8VC, and other leading investors.

uBiomes mission is to explore important research questions about the microbiome and to develop accurate and reliable clinical tests based on the microbiome.

Contact:Julie Taylorjulie(at)ubiome(dot)comPh: +1 (415) 212-9214

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uBiome Grant Will Enable UCSF Scientist to Explore 'Spit Test' to Predict Blood Infections in Young Bone Marrow ... - PR Web (press release)

In 2002, Ian Thompson, a specialist in facial reconstruction at Kings College, London, received an urgent phone call. A patient in his late 20s had been struck by an out-of-control car mounting the pavement. The impact had sent him catapulting over the bonnet of the car, smashing his face and shattering the fragile orbital floor the tiny bone, no more than 1mm thick, which holds the eyeball in place in the skull.

Without the orbital floor, your eye moves backwards into the skull, almost as a defensive mechanism, Thompson explains. But this results in blurred vision and lack of focus. This patient had also lost the ability to perceive colour. His job involved rewiring aircraft and as he could no longer detect a red wire from a blue one, hed barely been able to work in three years.

The accident had happened three years earlier. Since then, surgeons had desperately tried to reconstruct the bony floor and push the eye back into position, first using material implants and then bone from the patients own rib. Both attempts had failed. Each time, infection set in after a few months, causing extreme pain. And now the doctors were out of ideas.

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Thompsons answer was to build the worlds first glass implant, moulded as a plate which slotted in under the patients eye into the collapsed orbital floor. The idea of using glass a naturally brittle material to repair something so delicate may seem counterintuitive.

But this was no ordinary glass.

If you placed a piece of window glass in the human body, it would be sealed off by scar tissue, basically wobble around in the body for a while and then get pushed out, says Julian Jones, an expert in bioglass at Imperial College London. When you put bioglass in the body, it starts to dissolve and releases ions which kind of talk to the immune system and tell the cells what to do. This means the body doesnt recognise it as foreign, and so it bonds to bone and soft tissue, creating a good feel and stimulating the production of new bone.

Bioglass actually works even better than the patients own bone Ian Thompson

For Thompson, the results were immediate. Almost instantaneously, the patient regained full vision, colour and depth perception. Fifteen years on, he remains in full health.

Thompson has gone on to use bioglass plates to successfully treat more than 100 patients involved in car or motorcycle accidents. Bioglass actually works even better than the patients own bone, Thompson says. This is because weve found that it slowly leaches sodium ions as it dissolves, killing off bacteria in the local environment. So, quite by chance, you have this mild antibiotic effect which eliminates infections.

Cutting edge

Bioglass was invented by US scientist Larry Hench in 1969. Hench was inspired by a chance conversation on a bus with an army colonel who recently had returned from the Vietnam War. The colonel told Hench that while modern medical technology could save lives on the battlefield, it could not save limbs. Hench decided to shelve his research into intercontinental ballistic missiles and instead work on designing a bionic material which would not be rejected by the human body.

Hench ultimately took his research to London, and it has been in Britain where some of the most revolutionary bioglass innovations are being made in fields from orthopaedic surgery to dentistry.

Over the last 10 years, surgeons have used bioglass in a powdered form, which looks and feels like a gritty putty, to repair bone defects arising from small fractures. Since 2010, this same bioglass putty has hit the high street as the key component in Sensodynes Repair and Protect toothpaste, the biggest global use of any bioactive material. During the brushing process, the bioglass dissolves and releases calcium phosphate ions which bond to tooth mineral. Over time, they slowly stimulate regrowth.

But many scientists feel that the current applications of bioglass are barely scratching the surface of what could be possible. New clinical products are being developed which could revolutionise bone and joint surgery like never before.

Sitting in his office in Imperial Colleges Department of Materials, Jones is holding a small, cube-shaped object hes dubbed bouncy bioglass. Its similar to the current bioglass but with a slight twist: subtle alterations in the chemical composition mean its no longer brittle. Instead it bounces,like a kids power ball as Jones describes it, and its incredibly flexible.

The point of this is that it can be inserted into a badly broken leg and can support both the patients weight and allow them to walk on it without crutches, without requiring any additional metal pins or implants for support. At the same time, the bouncy bioglass also will stimulate and guide bone regrowth while slowly, naturally assimilating into the body.

To regenerate large pieces of bone, for example in a really big fracture, its very important to be able to put weight on your leg, Jones says. And its really important that the bio-implant in your leg is able to transmit the force from your weight to the bone cells, like a signal. Our body makes its own bone in the architecture that its in, because the cells feel the mechanical environment. So to grow back a big piece of bone you need to be able to transmit the right signals to them. The reason why astronauts in space lose bone mass is because without gravity, the cells arent receiving the same information as they do on Earth.

Further alterations to the chemical makeup of bioglass produce a different form which is much softer and has an almost rubbery feel. It feels almost like a piece of squid at a seafood restaurant. This bioglass is designed for possibly the holy grail of orthopaedic surgery: cartilage repair.

Right now, surgeons attempt to repair damaged cartilage in arthritic hips or damaged knee joints with a fiddly procedure called microfracture. This involves smoothing over the damaged area to expose the bone underneath, then pricking it to release stem cells from the bone marrow which stimulate repair. But this results in scar cartilage and within a few years, as many athletes have found, the original problem returns.

As a solution, Jones is looking to produce bioglass which can be 3D-printed and then slotted into any hole in the cartilage. For the cells to accept it, the material must retain all the natural properties of cartilage. To test its effectiveness, Jones uses a simulator that has human knee joints from cadavers donated for medical research.

We simulate the walking action, bending, all the things a knee would do, and make sure that the bioglass actually preserves the rest of the joint and behaves as it should do, he says. If that works then well proceed to animal and then clinical trials.

This same bioglass could find an additional use in aiding people with chronic back pain due to herniated discs. At the moment surgeons treat this by replacing the dysfunctional disc with a bone graft which fuses the vertebrae in the back together. But while this takes away the pain, it results in a considerable loss in mobility. Instead, a bioglass implant could be printed and simply inserted to replace the faulty disc.

It seems the obvious thing to do, Jones says. So far nobody has been able to replicate the mechanical properties of cartilage synthetically. But with bioglass, we think we can do it.

Weve just got to prove that we can. If all goes well and we pass all the necessary safety tests, it could reach the clinic in 10 years.

Using man-made materials which can fuse to the body may seem far-fetched but it is appearing to be a more and more likely component of future medicine. Already, millions of people brush their teeth with it. And that may just be the start.

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The best way to fix broken bones might be with glass - BBC News

For cancer patients, understanding the odds of a treatments success can be bewildering. The same drug, applied to the same type of cancer, might be fully successful on one persons tumour and do nothing for another one. Physicians are often unable to explain why.

Now, U of T Engineering researchers are beginning to understand one of the reasons.Abdullah Syed and Shrey Sindhwani, both PhD candidates,and their colleagues at the Institute of Biomaterials & Biomedical Engineering (IBBME) have created a technology to watch nanoparticles traveling into tumours revealing barriers that prevent their delivery to targets and the variability between cancers.

The biggest thing weve noticed is that nanoparticles face multiple challenges posed by the tumour itself on their way to cancer cells, says Sindhwani, an MD-PhD student in the Integrated Nanotechnology & Biomedical Sciences Laboratory of Professor Warren Chan (IBBME). Syed and Sindhwani co-published their findings online June 22, and on the cover of the Journal of the American Chemical Society. So the treatment might work for a while or worse, theres just enough of the drug for the cancer to develop resistance. This could be prevented if we can figure out the ways in which these barriers stop delivery and distribution of the drug throughout the cancer.

Tiny nanoparticles offer great hope for the treatment of cancer and other disease because of their potential to deliver drugs to targeted areas in the body, allowing more precise treatments with fewer side effects. But so far the technology hasnt lived up to its promise, due to delivery and penetration problems.

To dismantle this roadblock, the two graduate students searched for a way to better view the particles journey inside tumours. They discovered that the tough-to-see particles could be illuminated by scattering light off their surfaces.

The sensitivity of our imaging is about 1.4 millionfold higher, says Syed. First, we make the tissue transparent, then we use the signal coming from the particles to locate them. We shine a light on the particles and it scatters the light. We capture this scattering light to learn the precise location of the nanoparticles.

It was already understood that nanoparticles were failing to accumulate in tumours, thanks to a meta-analysis of the field done by Chans group. But the researchers have developed technologies to look at nanoparticle distribution in 3D, which provides a much fuller picture of how the particles are interacting with the rest of the tumour biology. The goal is to use this technology to gather knowledge for developing mathematical principles of nanoparticle distribution in cancer, similar to the way principles exist for understanding the function of the heart, says Syed.

And because each tumour is unique, this technology and knowledge base should help future scientists to understand the barriers to drug delivery on a personalized basis, and to develop custom treatments.

The next step is to understand what in cancers biology stops particles from fully penetrating tumours and then to develop ways to bypass cancers defences.

But the technology is also useful for diseases other than cancer. With the help of Professor Jennifer Gommerman, an researcher in the Department of Immunology who studies multiple sclerosis (MS), Syed and Sindhwani captured 3D images of lesions in a mouse model mimicking MS using nanoparticles.

This is going to be very valuable to anyone trying to understand disease or the organ system more deeply, says Sindhwani. And once we understand barriers that dont allow drugs to reach their disease site, we can start knocking them down and improving patient health adds Syed.

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Targeting tumours: IBBME researchers investigate biological barriers to nanomedicine delivery - U of T Engineering News

Empas multicellular model, which is mimicking the placental barrier: a core of connective tissue cells, surrounded by trophoblast cells. Credit: Empa

An Empa team has succeeded in developing a new three-dimensional cell model of the human placental barrier. The "model organ" can quickly and reliably deliver new information on the intake of substances, such as nano-particles, by the placental barrier and on any possible toxic effects for the unborn child. This knowledge can also be used in the future for the development of new approaches to therapy during pregnancy.

During its development, the foetus is extremely susceptible to toxic substances. Even the tiniest doses can cause serious damage. In order to protect the unborn child,one of the tasks of the placenta is to act as a barrier to "filter out" harmful substances, while at the same time providing the foetus with the nutrients it needs. In recent years, however, evidence has increasingly suggested that the placental barrier is not 100 percent effective and that nano-particles are actually able to penetrate it.

Nano-particles are being used in ever more varied areas of our lives. They are used, for example, in sun creams to protect against sunburn; they are used in condiments to stop them getting lumpy; they are used to make outdoor clothing waterproof and they are likely to be used in the future to transport medicines to their rightful destinations in the body . "At the moment, pregnant women are not being exposed to problematic amounts of nano-particles, but in the future that could well happen due to the ever increasing use of these tiny particles," suggests Tina Buerki of the "Department of Particles-Biology Interactions."

In order to ensure the safe development of nano-particles in the most diverse areas of application, their absorption mechanism at the placental barrier and their effect on the mother, foetus and placenta itself must be looked at more closely. It is the size, charge, chemical composition and shape of the nano-particles that could have an influence on whether they actually penetrate the placental barrier and, if so, in what way they are able to do so. At the moment, however, this research is only in its infancy. Since the function and structure of the human placenta is unique, studies undertaken on pregnant mammals are problematic and often inconclusive. Traditional models of the human placental barrier are either very time consuming to construct, or are extremely simplified.

A 3-D model of the human placental barrier

Tests of this nature are best carried out on donated placentas that become available after childbirth by Caesarean section. The organs are connected as quickly as possible to a perfusion system and this ensures the tissue is provided with nutrients and oxygen. This model is, indeed, the most accurate, i.e. the most clinically relevant. It is, however, very technically demanding and, moreover,restricted to a perfusion time window of six to eight hours. Against that, such placentas can be used to reliably test the ability of any given nano-particle to penetrate the placental barrier. The model does not, however, yield any information on the mechanism used by the particle to penetrate this complex organ.

Researchers are therefore tending to fall back on the use of simple cell cultures and other modelling systems. An individual cell, possibly taken from the epithelium and subsequently cultivated and propagated in a petri dish, is perfectly suited to a whole range of different experiments. However, researchers cannot be certain that the cells in the petri dish will ultimately behave like those in the human body. The new model that the Empa team under Tina Buerki described in the scientific journal Nanoscale at the end of last year is, by contrast, three-dimensional and consists of more than one cell type. The cells exist in a tissue-like environment analogous to the placenta and can be experimented on for a longer period of time.

Golden test candidate

In order to create the model, the research team used the "hanging drop" technology developed by Insphero AG. This technology allows models to be created without "scaffolding," which can hinder free access of the nano-particles to the cells in the subsequent transport tests. Rather than introducing the cells in a flat petri dish, a special device, in which the cells in the hanging drops combine to form spherical micro-tissue, is used. The resulting micro-tissue mimics the human placenta much more closely than cells cultivated on a "rigid" culture dish. Experiments can be carried out much more quickly using the 3-D model than with the real placenta and, significantly, on the most widely differing types of nano-particle. In this way, those nano-particles that show potentially toxic effects or demonstrate desirable transport behaviour can be efficiently pre-selected and the results verified using a real placenta.

The model has already proved itself in a second study, which the team has just published in the scientific journal Nanomedicine. Buerki's team has come up with an absorption mechanism for gold particles that could be used in a range of medicinal applications. The Empa team looked at gold particles of various sizes and different surface modifications. In accordance with the results of other studies, the researchers discovered that small gold particles were able to penetrate the placental barrier more easily. In addition, fewer particles passed through the barrier if they were carrying polyethylene glycol (PEG) on their surfaces. These are chain-forming molecules that almost completely envelope the particles. PEG is often used in medicine to allow particles and other small structures to travel "incognito" in the body, thus preventing them being identified and removed by the immune system. "It therefore appears possible to control the movement of nano-particles through the placenta by means of their properties," Buerki explains.

Medicines for pregnant women that do not harm the child

Empa's research team is keen to further develop this 3-D model in the future. The team is hoping to augment the model using a dynamic component. This would, for example, mean introducing the micro-tissue in a micro-fluid system able to simulate blood circulation in the mother and child. Another approach would be to combine the model of the placenta with other models. "With the model of a foetus, for example," Buerki suggests. In this way, complex organ interactions could also be incorporated and it would be possible, for example, to discover whether the placenta releases foetus-damaging substances as a reaction to certain nano-particles.

"With these studies, we are hoping to lay the foundations for the safe but nevertheless effective use of nano-medicines during pregnancy," Buerki continued. If we understand the transport mechanisms of nano-materials through the placental barrier well enough, we believe we can develop new carrier systems for therapeutic agents that can be safely given to pregnant women. This is because many women are forced to take medicines even during pregnancy patients suffering from epilepsy or diabetes, for example, or patients that have contracted life-threatening infections. Nano-carriers must be chosen which are unable to penetrate the placental barrier. It is also possible, for example, to provide such carriers with "address labels," which ensure that the medicine shuttle is transported to the correct organ i.e. to the diseased organ and is unable to penetrate the placenta. This would allow the medicine to be released first and foremost into the mother. Consequently, the amounts absorbed by the foetus or embryoand therefore the risk to the unborn child are significantly reduced.

Explore further: New placenta model could reveal how birth defect-causing infections cross from mom to baby

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Medication for the unborn baby - Medical Xpress

By Ben Hirschler

LONDON (Reuters) - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.

Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States.

Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases.

After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.

In the past five years, European regulators have approved two gene therapies - the first of their kind in the world, outside China - but only three patients have so far been treated commercially.

UniQure's Glybera, for a very rare blood disorder, is now being taken off the market given lack of demand.

The future of GlaxoSmithKline's Strimvelis for ADA-SCID - or "bubble boy" disease, where sufferers are highly vulnerable to infections - is uncertain after the company decided to review and possibly sell its rare diseases unit.

Glybera, costing around $1 million per patient, has been used just once since approval in 2012. Strimvelis, at about $700,000, has seen two sales since its approval in May 2016, with two more patients due to be treated later this year.

"It's disappointing that so few patients have received gene therapy in Europe," said KPMG chief medical adviser Hilary Thomas. "It shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment."

These first two therapies are for exceptionally rare conditions - GSK estimates there are only 15 new cases of ADA-SCID in Europe each year - but both drugs are expected to pave the way for bigger products.

The idea of using engineered viruses to deliver healthy genes has fuelled experiments since the 1990s. Progress was derailed by a patient death and cancer cases, but now scientists have learnt how to make viral delivery safer and more efficient.

Spark Therapeutics hopes to win U.S. approval in January 2018 for a gene therapy to cure a rare inherited form of blindness, while Novartis could get a U.S. go-ahead as early as next month for its gene-modified cell therapy against leukaemia - a variation on standard gene therapy.

At the same time, academic research is advancing by leaps and bounds, with last week's successful use of CRISPR-Cas9 gene editing to correct a defect in a human embryo pointing to more innovative therapies down the line.

Spark Chief Executive Jeffrey Marrazzo thinks there are specific reasons why Europe's first gene therapies have sold poorly, reflecting complex reimbursement systems, Glybera's patchy clinical trials record and the fact Strimvelis is given at only one clinic in Italy.

He expects Spark will do better. It plans to have treatment centers in each country to address a type of blindness affecting about 6,000 people around the world.

Marrazzo admits, however, there are many questions about how his firm should be rewarded for the $400 million it has spent developing the drug, given that healthcare systems are geared to paying for drugs monthly rather than facing a huge upfront bill.

A one-time cure, even at $1 million, could still save money over the long term by reducing the need for expensive care, in much the same way that a kidney transplant can save hundreds of thousands of dollars in dialysis costs.

But gene therapy companies - which also include Bluebird Bio, BioMarin, Sangamo and GenSight - may need new business models.

One option would be a pay-for-performance system, where governments or insurers would make payments to companies that could be halted if the drug stopped working.

"In an area like haemophilia I think that approach is going to make a ton of sense, since the budget impact there starts to get more significant," Marrazzo said.

Haemophilia, a hereditary condition affecting more than 100,000 people in markets where specialty drugmakers typically operate, promises to be the first really big commercial opportunity. It offers to free patients from regular infusions of blood-clotting factors that can cost up to $400,000 a year.

Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta-thalassaemia, another inherited blood disorder.

Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene-modified cell therapies reached $1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.

Shire CEO Flemming Ornskov - who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder - sees both the opportunities and the difficulties of gene therapy.

"Is it something that I think will take market share mid- to long-term if the data continues to be encouraging? Yes. But I think everybody will have to figure out a business model."

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Gene Therapy Is Now Available, but Who Will Pay for It? - Scientific American