Monthly Archives: February 2017

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.

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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