Category Archives: Gene Medicine

For decades, some unluckydog lovers have welcomeda bundle of barking joy into their homes, only to see them perish from a mysterious disease mere weeks after their birth. The pups seemingly healthy muscles had literally wasted away in front of their owners eyes until they could no longer stand and breathe.

It wasnt until 2010 that a French research team isolated the genetic cause of this specific muscle-wasting disease in a group of Labrador Retrievers; these dogs were suffering from a single mutation that left them unable to produce an essential protein known asmyotubularin.Whats more, it was the exact kind of mutation and disease also long found in male human babies, too. That made the researchers wonder if these unfortunate puppiescould help us study the disease and even someday find a way to saveboth pets and people.

Now, years down the road, it appearsthey were right, thanks to a cutting-edgegene therapy treatment.

An international group of researchers, including some from the original French team, gathered together 10-week-old puppies with the mutation to take part in a randomized controlled trial. The dogs who were given a treatment that repaired their defectivemyotubularingene avoided the crippling muscle degeneration that killed the placebo-treated dogs by week 17. And by the ninth month of study, the saved puppies muscle and neurological function continued to match readings from healthy dogs, particularly forthose that got the highest doses.

The findings, building on an earlier proof-of-concept study of dogs and mice by the researchers, signal that a scaled-up treatment could save the lives of boys with the same sort of genetic flaw.

I believe that the dog study will be about as close as we will ever get to a human study, senior author Dr. Martin Childers of the University of Washington told Vocativ in an email. Because we found evidence that the gene therapy product spread throughout the entire skeletal musculature of adult dogs after a single infusion, it seems reasonable to expect a similar result in human patients.

Gene therapy has received plenty of attention for its potential to treat otherwise irreparable DNA defects, but according to the researchers, theres been little focus on bone- and muscle-relatedgenetic disorders. The condition treated in the current study, called x-linked myotubular myopathy, affects around one in every 50,000 boys, with most sufferers living no more than a few years. And though theres no true tally of how often it affects dogs, case reports of similar-sounding diseases have been published stretching back decades.

There will undoubtedly be hurdles to climb before the treatment Childers and his team developed, or a similar one, can be tested in people, Childers said. It is always possible that humans might respond differently, thus, clinical trials will be conducted with extraordinary care and oversight, he explained. And though the dogs suffered little adverse effects from the therapy delivered via a harmless virus researchers will still have to watch out for any possible toxicity in people.

That said, the treatment offers hope for both man and mutts. The changes seen after a single treatment have lasted for several years in the small sample of dogs the team has raised. So its possible that people wont need repeated doses or they would be infrequent, Childers said a big positive, given how expensive gene therapy is today.

And its also likely that these treatments, within the larger field of regenerative medicine, will find a place for dogs and other animals sooner than it will for people.

Veterinary medicine is ahead of human medicine in some cases with respect to regenerative technologies, Childers said. Stem cell infusions, for example, have been given to pets and horses for more than a decade.

But people may not have to wait so long for the promise of gene therapy either. Childers is hopeful that Audentes Therapeutics, a San Francisco biomedical company hes collaborating with (and which partially funded the current study), will begin their first human trials of a gene therapy treatment for x-linked myotubular myopathy, based on his teams research, later this year.

The teams findings were published earlier this February in Molecular Therapy.

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Gene Therapy Saves Puppies From A Fatal DiseaseAnd Maybe Us Next - Vocativ

Personalized medicine, which involves tailoring health care to each persons unique genetic makeup, has the potential to transform how we diagnose, prevent and treat disease. After all, no two people are alike. Mapping a persons unique susceptibility to disease and targeting the right treatment has deservedly been welcomed as a new power to heal.

The human genome, a complete set of human DNA, was identified and mapped a decade ago. But genomic science remains in its infancy. According to Francis Collins, the director of the National Institutes of Health, It is fair to say that the Human Genome Project has not yet directly affected the health care of most individuals.

Its not that there havent been tremendous breakthroughs. Its just that the gap between science and its ability to benefit most patients remains wide. This is mainly because we dont yet fully understand the complex pathways involved in common chronic diseases.

I am part of a research team that has taken on the ambitious goal of narrowing this gap. New technologies are allowing us to probe DNA, RNA, proteins and gut bacteria in a way that will change our understanding of health and disease. Our hope is to discover novel biological markers that can be used to diagnose and treat common chronic conditions, including Alzheimers disease, heart disease, diabetes and cancer.

But when it comes to preventing the leading causes of death which include chronic diseases, genomics and precision medicine may not do as much as we hope.

Many diseases arent due only to genetics

Chronic diseases are only partially heritable. This means that the genes you inherit from your parents arent entirely responsible for your risk of getting most chronic diseases.

The estimated heritability of heart disease is about 50 percent. Its 64 percent for Type 2 diabetes mellitus, and 58 percent for Alzheimers disease. Our environment and lifestyle choice are also major factors; they can change or influence how the information coded in our genes is translated.

Chronic diseases are also complex. Rather than being controlled by a few genes that are easy to find, they are weakly influenced by hundreds if not thousands of genes, the majority of which still elude scientists. Unlocking the infinite combinations in which these genes interact with each other and with the environment is a daunting task that will take decades, if ever, to achieve.

While unraveling the genomic complexity of chronic disease is important, it shouldnt detract from existing simple solutions. Many of our deadliest chronic diseases are preventable. For instance, among U.S. adults, more than 90 percent of Type 2 diabetes, 80 percent of coronary arterial disease, 70 percent of stroke and 70 percent of colon cancer are potentially avoidable.

Smoking, weight gain, lack of exercise, poor diet and alcohol consumption are all risk factors for these conditions. Based on their profound impact on gene expression, or how instructions within a gene are manifested, addressing these factors will likely remain fundamental in preventing these illnesses.

Will more knowledge be more power?

A major premise behind personalized medicine is that empowering patients and doctors with more knowledge will lead to better decision-making. With some major advances, this has indeed been the case. For instance, variants in genes that control an enzyme that metabolizes drugs can identify individuals who metabolize some drugs too rapidly (not giving them a chance to work), or too slowly (leading to toxicity). This can lead to changes in medication dosing.

When applied to prevention, however, identifying our susceptibility at an earlier stage has not aided in avoiding chronic diseases. Research challenges the assumption that we will use genetic markers to change our behavior. More knowledge may nudge intent, but that doesnt translate to motivating changes to our lifestyle.

A recent review found that even when people knew their personal genetic risk of disease, they were no more likely to quit smoking, change their diet or exercise. Expectations that communicating DNA-based risk estimates changes behavior is not supported by existing evidence, the authors conclude.

Increased knowledge may even have the unintended consequence of shifting the focus to personal responsibility while detracting from our joint responsibility for improving public health. Reducing the prevalence of chronic diseases will require changing the political, social and economic environment within which we make choices as well as individual effort.

What about treating chronic diseases?

Perhaps the most awaited hope of the genomic era is that we will be able to develop targeted treatments based on detailed molecular profiling. The implication is that we will be able to subdivide disease into new classifications. Rather than viewing Type 2 diabetes as one disease, for example, we may discover many unique subtypes of diabetes.

This already is happening with some cancers. Patients with melanoma, leukemia or metastatic lung, breast or brain cancers can, in some cases, be offered a molecular diagnosis to tailor their treatment and improve their chance of survival.

We have been able to make progress in cancer therapy and drug safety and efficacy because specific gene mutations control a persons response to these treatments. But for complex, chronic diseases, relatively few personalized targeted treatments exist.

Customizing treatments based on our uniqueness will be a breakthrough, but it also poses a challenge: Without the ability to test targeted treatments on large populations, it will make it infinitely harder to discover and predict their response.

The very reason we group people with the same signs and symptoms into diagnoses is to help predict the average response to treatment. There may be a time when we have one-person trials that custom tailor treatment. However, the anticipation is that the timeline to getting to such trials will be long, the failure rate high and the cost exorbitant.

Research that takes genetic risk of diabetes into account has found greater benefit in targeting prevention efforts to all people with obesity rather than targeting efforts based on genetic risk.

We also have to consider decades of research on chronic diseases that suggest there are inherent limitations to preventing the global prevalence of these diseases with genomic solutions. For most of us, personalized medicine will likely complement rather than replace one-size-fits-all medicine.

Where does that leave us? Despite the inherent limitations to the ability of genomic medicine to transform health care, medicine in the future should unquestionably aspire to be personal. Genomics and molecular biosciences will need to be used holistically in the context of a persons health, beliefs and attitudes to fulfill their power to greatly enhance medicine.

Sharon Horesh Bergquist, Physician, teacher, researcher in preventive medicine and healthy aging, Emory University

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Personalized medicine may do more to treat rather than prevent chronic diseases - Salon

February 20, 2017 Credit: CC0 Public Domain

Gene editing techniques developed in the last five years could help in the battle against cancer and inherited diseases, a University of Exeter scientist says.

Dr Edze Westra said the ability to splice selected DNA into cells with great precision would become "super important" in the next two decades. There could be benefits for generations of people affected by cancer, failing vision and the diseases of old age or bad genes.

"There is always a risk with this kind of technology and fears about designer babies and we have started having discussions about that so we can understand the consequences and long-term risks," said Dr Westra, of the Environment and Sustainability Institute on the University of Exeter's Penryn Campus in Cornwall. "I think in the coming decades gene editing will become super important, and I think we will see it being used to cure some inherited diseases, to cure cancers, to restore sight to people by transplanting genes. I think it will definitely have massive importance."

On Tuesday, two highly influential academic bodies in the US shook up the scientific world with a report that, for the first time, acknowledged the medical potential of editing inherited genes. The National Academy of Sciences and National Academy of Medicine ruled that gene editing of the human "germline"eggs, sperm and embryosshould not be seen as a red line in medical research.

Many critics insist that powerful new gene editing techniques should never be used to alter inherited DNA. They argue that such a move would be the start of a slippery slope leading to "designer" babies with selected features such as blue eyes, high intelligence or sporting prowess.

But the two pillars of the American scientific establishment said that with necessary safeguards, future use of germline gene editing to treat or prevent disease and disability was a "realistic possibility that deserves serious consideration".

Dr Westra is taking part in a discussion on gene editing and its potential implications for society at the American Association for the Advancement of Science (AAAS) annual meeting in Boston, Massachusetts. He said gene editing technology not only held out the promise of fixing genetic faults, but could be used to turn cells into miniature factories that churned out therapeutic chemicals or antibodies.

One application was the use of "gene drives" that increase the prevalence of a certain trait in a population. For instance, gene editing machinery placed inside the cells of large numbers of malaria transmitting mosquitoes could prevent them spreading the organism that causes the disease to humans.

The most promising form of gene editing, known as CRISPR/Cas9, was first demonstrated in 2012. It employs a defence system bacteria use to protect themselves against viruses. A carefully targeted enzyme is used as chemical "scissors" that cut through specific sections of double stranded DNA. Then the cell's own DNA repair machinery can be exploited to insert the "pasted" genetic material.

Dr Westra said: "Gene editing is causing a true revolution in science and medicine because it allows for very precise DNA surgery. "A mutation in a gene that causes disease can now be repaired using CRISPR."

Explore further: No designer babies, but gene editing to avoid disease? Maybe

Gene editing techniques developed in the last five years could help in the battle against cancer and inherited diseases, a University of Exeter scientist says.

(Medical Xpress)A team of researchers with New England Biolabs Inc. (NEB) has found that sequenced DNA samples held in public databases had higher than expected low-frequency mutation error rates. In their paper published ...

Personalized medicine, which involves tailoring health care to each person's unique genetic makeup, has the potential to transform how we diagnose, prevent and treat disease. After all, no two people are alike. Mapping a ...

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative ...

A genomic study of baldness identified more than 200 genetic regions involved in this common but potentially embarrassing condition. These genetic variants could be used to predict a man's chance of severe hair loss. The ...

Purdue University and Indiana University School of Medicine scientists were able to force an epigenetic reaction that turns on and off a gene known to determine the fate of the neural stem cells, a finding that could lead ...

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Gene editing could help tackle cancer and inherited diseases - Medical Xpress

The 1997 film Gattaca predicted a near future in which cities are powered by vast solar thermal arrays, humans launch manned missions to Saturns moons, and doctors design super smart and strong babies. A generation later, it is the gene editing that is proving most prescient.

Over the past decade, huge advances in gene-editing techniques have enabled researchers to slice up and rewrite DNA with incredible precision. At the forefront of the ensuing revolution is the CRISPR-Cas9, a technology derived from bacteria that enables scientists to snip and repair DNA, nucleotide by nucleotide, quickly and cheaply. The potential uses are vast. And so are the ethical quandaries.

The National Academies of Sciences and the National Academy of Medicine convened a panel to recommend guidelines for the use of powerful gene-editing tools. The results, released this week, are thoughtful and should for the moment, anyway channel research and testing in unambiguously positive directions.

CRISPR can be used in basic laboratory research, revealing how disease works on the molecular level. This is similar enough to other types of lab research that it requires no novel scientific or ethical standards. Researchers can also treat live humans with gene editing technologies, for example by taking immune cells out of the body, altering them and re-inserting them to fight an advanced cancer. Therapies such as these are already under development, and although researchers have to be cautious about off-target gene slicing, existing rules governing the development of medical treatments should suffice.

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The ethics get much trickier when researchers want to change the DNA in reproductive cells, which would alter the genes that parents pass to children, forever. Doing so could prevent vast amounts of human suffering. But there is a problem of consent: Future generations have no say in their alteration. Disability communities would no doubt feel threatened and stigmatized, because gene editing could be used to essentially remove their type from the gene pool. Changes made to enhance human offspring, rather than simply to combat disease and disability, could redefine what it means to be human, while those to whom these techniques are unavailable would risk becoming a genetic underclass. A line would have to be drawn between heritable changes that are clearly valuable and those that risk unnecessarily humiliating people, destabilizing society and changing the nature of humanity.

The panel attempted to draw a preliminary line and put it in the right place. Heritable changes should be attempted only when scientists are convinced that specific genes cause or strongly predispose people to getting a serious disease or a condition, and when they know what normal genetic code should look like. They should only intervene when there are no reasonable alternatives available to families, and when real-world evidence shows that the benefits outweigh the risks.

The debate will not and should not end there. But before society has a full chance to process these questions, the panels approach is the right one. The goal should be to stop crippling diseases, not to build designer babies.

Editorial courtesy of the Washington Post.

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Editorial: A way forward in gene editing - New Haven Register

New gene drive technology carries hope and risk

Caroline Davis2010 / Flickr

By Yasemin SaplakogluFeb. 19, 2017 , 12:15 PM

Q: Should we be looking at how the environment might be affected by gene drives?

A: Absolutely, this is a manipulation of nature. We dont know how it would affect population dynamics and ecosystems. In some cases, the purpose of gene drives would be to reduce population sizes of an organism, which could influence processes like pollination and transmission of parasites. In other cases, we would use gene drives to weed out disease by driving the population that carries that disease to extinction.

Q: What is the worst-case scenario of releasing these organisms?

A: Eliminating an organism or reducing its numbers greatly. By eliminating one plant species, you cause the proliferation of others, and this leads to a series of changes in the ecosystem. We need to understand the system well enough so that we can take ethical concerns into account as we make decisions.

Evolutionary ecologist James Collins

Charles Kazilek

Q: Who gets to make these decisions?

A:Social scientists are trying to come up with better ways to sample human populations to get a better sense of whats tolerable and whats not tolerable in terms of their release. . If you release [modified mosquitoes] in Town A, the mosquitoes may not have any problem flying to Town B, even though Town B is not interested in having them. Theyll go anyway.

A:The advantage of these other technologies is that they are effective only as long as youre releasing modified male mosquitoes. When you stop the manipulation, the population would bounce back to normal levels. You have a control over the system that is yet to be demonstrated for gene drives where once you alter the genes in these populations, they just keep changing.

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New gene drive technology could wipe out malaria, but is it safe? - Science Magazine

Vitamin B3, also known as niacin and nicotinic acid, prevents eye degeneration in glaucoma-prone mice, according to a study published in the Feb. 17 issue of the journal Science.

Williams et al show that dietary supplementation with a single molecule (vitamin B3 or NAM) or Nmnat1 gene therapy significantly reduces vulnerability to glaucoma by supporting mitochondrial health and metabolism. Image credit: Mizianitka.

Glaucoma, a group of complex, multifactorial diseases, is one of the most common neurodegenerative diseases worldwide and the most common cause of age-related blindness in the United States. There is currently no cure, and once vision is lost, the condition is irreversible.

In most glaucoma patients, harmfully high pressure inside the eye or intraocular pressure leads to the progressive dysfunction and loss of retinal ganglion cells (neuronal cells that connect the eye to the brain via the optic nerve).

Increasing age is a key risk factor for glaucoma, contributing to both harmful elevation of intraocular pressure and increased neuronal vulnerability to pressure-induced damage.

We wanted to identify key age-related susceptibility factors that change with age in the eye and increase vulnerability to disease and in particular neuronal disease, said Prof. Simon W.M. John, from the Jackson Laboratory, Tufts University of Medicine and the Howard Hughes Medical Institute.

By understanding general age-related mechanism, there is the potential to develop new interventions to generally protect from common age-related disease processes in many people.

Conducting a variety of genomic, metabolic, neurobiological and other tests in DBA/2J mice, a widely used model of chronic, age-related, inherited glaucoma, Prof. John and co-authors discovered that NAD a molecule vital to energy metabolism in neurons and other cells declines with age.

The decrease in NAD levels reduces the reliability of neurons energy metabolism, especially under stress such as increased intraocular pressure.

Like taking that big hill on your old bike, some things are going to fail more often, said Prof. John, corresponding author of the study.

The amount of failure will increase over time, resulting in more damage and disease progression.

In essence, the treatments of vitamin B3 boosted the metabolic reliability of aging retinal ganglion cells, keeping them healthier for longer.

Because these cells are still healthy, and still metabolically robust, even when high intraocular pressure turns on, they better resist damaging processes, said Dr. Pete Williams, first author of the study and a researcher at the Jackson Laboratory.

The researchers also found that a single gene-therapy application of Nmnat1 the gene for an enzyme that makes NAD from nicotinamide prevented glaucoma from developing in DBA/2J mice.

It can be a problem for patients, especially the elderly, to take their drugs every day and in the correct dose. So gene therapy could be a one-shot, protective treatment, Dr. Williams said.

Gene therapies, through injections into the eye, have been approved for a handful of very rare, human genetic eye disorders, and their demonstration of an important age-dependent factor may enable gene therapy for more common eye disease.

The authors are pursuing clinical partnerships to begin the process of testing the effectiveness of vitamin B3 treatment in glaucoma patients. They are also exploring potential applications for the treatment in other diseases involving neurodegeneration.

_____

Pete A. Williams et al. 2017. Vitamin B3 modulates mitochondrial vulnerability and prevents glaucoma in aged mice. Science 355 (6326): 756-760; doi: 10.1126/science.aal0092

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Vitamin B3 Protects Mice from Glaucoma, Study Finds - Sci-News.com

WASHINGTON Dont expect designer babies any time soon but a major new ethics report leaves open the possibility of one day altering human heredity to fight genetic diseases, with stringent oversight, using new tools that precisely edit genes inside living cells.

Whats called genome editing already is transforming biological research, and being used to develop treatments for patients struggling with a range of diseases.

The science is nowhere near ready for a huge next step that raises ethical questions altering sperm, eggs or embryos so that babies dont inherit a disease that runs in the family, said a report from the National Academy of Sciences and National Academy of Medicine.

But if scientists learn how to safely pass alterations of the genetic code to future generations, the panel said germline editing could be attempted under strict criteria, including that it targets a serious disease with no reasonable alternative and is conducted under rigorous oversight.

Caution is absolutely needed, but being cautious does not mean prohibition, said bioethicist R. Alta Charo of the University of Wisconsin-Madison.

This committee is not saying we will or should do germline heritable editing. What we are saying is that we can identify a set of strict conditions under which it would be permissible to do it, Charo added. But we are far, far away from being ready to try.

Genome editing should not go beyond healing the sick and enhance traits such as physical strength, whats commonly called designer babies, the panel stressed.

But the public should get involved in these debates now, to say what might one day be acceptable.

The long-awaited report offers advice the prestigious academies cannot set policy.

But it is considered a step toward creating international norms for responsible development of this powerful technology.

The U.S. National Academies and its counterparts in Britain and China have been holding international meetings with the hope of doing just that.

Genome editing is a new tool for gene therapy and it has tremendous promise, Charo said.

But, she added, it has to be pursued in a way that promotes well-being and is responsible, respectful and fair.

Genome editing is essentially a biological version of cut-and-paste software, allowing scientists to turn genes on or off, repair or modify them inside living cells.

There are a few older methods but one with the wonky name CRISPR-Cas9 is so much faster, cheaper and simpler to use that it has spurred an explosion of research.

Under development are ways to treat a range of diseases from sickle cell and hemophilia to cancer. In lab experiments using human cells or animals engineered with humanlike disorders, scientists are unraveling how gene defects fuel disease and are even trying to grow transplantable human organs inside pigs.

That kind of research is very promising, is adequately regulated today and should continue at full speed, the National Academies panel concluded.

When it comes to the more sci fi-sounding uses, its quite possible scientists will learn how to perform germline editing in five to 10 years, said panel co-chair Richard Hynes of the Massachusetts Institute of Technology.

Safety is one reason for caution, he said, as scientists will have to learn whether editing one gene has unwanted downstream effects.

Some critics argue that families plagued by inherited diseases already have other alternatives adopt, use donated eggs, or undergo in vitro fertilization and discard resulting embryos that inherit the bad gene. But Charo noted that sometimes parents carry two copies of a lethal gene, guaranteeing any children inherit it. Others oppose the discarding of embryos for religious reasons.

For some families, you can see there would be strong arguments for doing it if the other criteria are met, said Robin Lovell-Badge of Britains Francis Crick Institute.

Some countries prohibit any germline editing research. Others, such as Britain, allow laboratory research with genome editing in embryos, not for pregnancy but to understand human development.

In the U.S., scientists can perform laboratory embryo research only with private, not government, funding. Any attempt at pregnancy would require permission from the Food and Drug Administration, which is currently prohibited from using federal funds to review any such request.

The bottom line is there is no planetary government with enforcement power, Charo noted.

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Could gene editing help prevent disease? Maybe - Mohave Valley News

All inherited diseases and cancers could be cured in the coming decades, according to a leading British expert.

Gene editing techniques that have been developed in recent years could be put to work to effectively end cancer and inherited diseases, according to DrEdze Westra

MrWestra believes that the ability to splice DNA into cells precisely a technology which is on the horizon, but is rejected on moral grounds by many will become super importantover the next 20 years.

It could completely transform the human race, he says so thatpeople are not affected by cancer, failing vision or the diseases of old age.

The bioscientist from the University of Exeter said: There is always a risk with this kind of technology and fears about designer babies and we have started having discussions about that so we can understand the consequences and long-term risks.

I think in the coming decades gene editing will become super important, and I think we will see it being used to cure all inherited diseases, to cure cancers, to restore sight to people by transplanting genes.

I think it will definitely have massive importance.

On Tuesday, two highly influential academic bodies in the US shook up the scientific world with a report that, for the first time, acknowledged the medical potential of editing inherited genes.

The National Academy of Sciences and National Academy of Medicine ruled that gene editing of the human germlineeggs, sperm and embryos should not be seen as a red line in medical research.

Many critics insist that powerful new gene editing techniques should never be used to alter inherited DNA.

They argue that such a move would be the start of a slippery slope leading to designerbabies with selected features such as blue eyes, high intelligence or sporting prowess.

But the two pillars of the American scientific establishment said that with necessary safeguards, future use of germline gene editing to treat or prevent disease and disability was a realistic possibility that deserves serious consideration.

Mr Westra is taking part in a discussion on gene editing and its potential implications for society at the American Association for the Advancement of Science (AAAS) annual meeting in Boston, Massachusetts.

He said gene editing technology not only held out the promise of fixing genetic faults, but could be used to turn cells into miniature factories that churned out therapeutic chemicals or antibodies.

One application was the use of gene drivesthat increase the prevalence of a certain trait in a population.

For instance, gene editing machinery placed inside the cells of large numbers of malaria transmitting mosquitoes could prevent them spreading the organism that carriesthe disease to humans.

It could be a fantastic strategy to deal with some of the worlds biggest problems,said Mr Westra.

In terms of ethics we need to work out what happens if a genetically engineered insect flies out of the window of the lab. Trials into gene drives are already happening in labs for malaria.

The most promising form of gene editing, known as CRISPR/Cas9, was first demonstrated in 2012.

It employs a defence system bacteria useto protect themselves against viruses.

A carefully targeted enzyme is used as chemical scissorsthat cut through specific sections of double stranded DNA. Then the cells own DNA repair machinery can be exploited to insert the pastedgenetic material.

Mr Westra said: Gene editing... is causing a true revolution in science and medicine, because it allows for very precise DNA surgery.

A mutation in a gene that causes disease can now be repaired using CRISPR.

PA

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Gene editing could bring an end to all inherited disease and cancer, expert says - The Independent

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Gene therapy treats muscle-wasting disease in dogs Science Daily Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative Medicine scientists are ... Gene therapy tried in dogs with muscle disease could prove useful for peopleFierceBiotech all 2 news articles »

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Gene therapy treats muscle-wasting disease in dogs - Science Daily

Although common, male baldness can have negative psychological effects and some studies have even linked it to a handful of serious illnesses. New research identifies the genetic variants involved in the condition, which could eventually enable researchers to predict a person's chances of hair loss.

Male baldness - also referred to as androgenetic alopecia or male pattern baldness (MPB) - affects a significant number of people in the United States, as the condition accounts for over 95 percent of all hair loss in men.

According to the American Hair Loss Association, two thirds of U.S. adults will be affected by MPB to a certain degree by the age of 35, and around 85 percent of men will have experienced significant hair loss by the age of 50.

A lot of these men are seriously affected by the condition, which can have a negative effect on a person's self-image, as well as on their interpersonal relationships.

Additionally, some genetic studies have even associated MPB with negative clinical outcomes such as prostate cancer and cardiovascular disease.

A new study - led by Saskia Hagenaars and David Hill of the University of Edinburgh in the United Kingdom - explores the genetic basis for the condition. The findings were published in the journal PLOS Genetics.

Scientists analyzed the genomic and health data of more than 52,000 men enrolled in the UK Biobank - an international health resource offering health information on more than 500,000 individuals.

The team located more than 250 independent genetic regions linked to severe hair loss.

The researchers split the 52,000 participants into two groups: a so-called discovery sample of 40,000 people and a target sample of 12,000 individuals. Based on the genetic variants that separated those with no hair loss from those with severe hair loss, the team designed an algorithm aimed to predict who would develop MPB.

The algorithmic baldness predictor is based on a genetic score, and although accurate predictions are still a long way off, the results of this study might soon enable researchers to identify subgroups of the population that are particularly prone to hair loss.

In the present study, researchers found that 14 percent of the participants with a submedian genetic score had severe MPB, and 39 percent had no hair loss. By contrast, 58 percent of those scoring in the top 10 percent on the polygenic score had moderate to severe MPB.

Co-lead author Saskia Hagenaars - a Ph.D. student at the University of Edinburgh's Centre for Cognitive Aging and Cognitive Epidemiology - comments on the findings:

"We identified hundreds of new genetic signals," Hagenaars says. "It was interesting to find that many of the genetics signals for male pattern baldness came from the X chromosome, which men inherit from their mothers."

The study's other lead author, Dr. David Hill, notes that the study did not collect data on the age of baldness onset, but only on hair loss pattern. However, he adds that, "we would expect to see an even stronger genetic signal if we were able to identify those with early-onset hair loss."

To the authors' knowledge, this is the largest genetic study of MPB to date.

The study's principal investigator, Dr. Riccardo Marioni, from the University of Edinburgh's Centre for Genomic and Experimental Medicine, explains the significance of the findings:

"We are still a long way from making an accurate prediction for an individual's hair loss pattern. However, these results take us one step closer. The findings pave the way for an improved understanding of the genetic causes of hair loss."

Learn how a drug promises robust new hair growth.

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Genetic basis for male baldness identified in large-scale study - Medical News Today