Showing posts with label Gene therapy. Show all posts
Showing posts with label Gene therapy. Show all posts

Friday, November 20, 2015

Biotech company Editas Medicine is planning to start human trials to genetically edit genes and reverse blindness


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The Telegraph

First genetically modified humans could exist within two years

Biotech company Editas Medicine is planning to start human trials to genetically edit genes and reverse blindness 


Humans who have had their DNA genetically modified could exist within two years after a private biotech company announced plans to start the first trials into a ground-breaking new technique.
Editas Medicine, which is based in the US, said it plans to become the first lab in the world to ‘genetically edit’ the DNA of patients suffering from a genetic condition – in this case the blinding disorder ‘leber congenital amaurosis’.
The disorder prevents normal function of the retina; the light-sensitive layer of cells at the back of the eye. It appears at birth or in the first months of life and eventually sufferers can go completely blind.
“Hereditary eye disease in an obvious place to start given that there is already precedent in classical gene therapy"
 
Professor Darren Griffin, University of Kent
 
The rare inherited disease is caused by defects in a gene which instructs the creation of a protein that is essential to vision.

But scientists at Editas Medicine in the US believe they can fix the mutated DNA using the ground-breaking gene-editing technology Crispr.

Katrine Bosley, the chief executive of Editas Medicine, told a conference in the US that the company hopes to start trialling the technology on blind patients in 2017.

It would be the first time the technology has been used on humans. Gene editing is currently banned in the US, so the company would need special permission from health regulators.

“It feels fast, but we are going at the pace science allows,” Bosley told the EmTech conference in Cambridge, Massachusetts.


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Wednesday, September 18, 2013

Next month, the US Food and Drug Administration will hold a two-day public meeting to discuss genetic modification within the human egg

FDA to hold first public hearing on GM babies


Rady Ananda
Actvist Post
Next month, the US Food and Drug Administration will hold a two-day public meeting to discuss genetic modification within the human egg, which changes will be passed on generationally.
Human gene therapy has been ongoing since 1990, but most of that involved non-heritable genes, called somatic (non-sex cell) gene therapy. Somatic modifications only affect the individual and are not passed on, and so do not affect the human genome.
The game changed with the successful birth of at least 30 genetically modified babies by 2001. Half of the babies engineered from one clinic developed defects and so the FDA stepped in and asserted jurisdiction over “the use of human cells that receive genetic material by means other than the union of gamete nuclei” (sperm and egg).
Now the FDA is considering going forward with “oocyte modification” which involves genetic material from a second woman, whereby offspring will carry the DNA from three parents. These kinds of genetic changes (“germline modification”) alter the human genome.
This is the first such meeting ever to be held in public by the FDA, reports Biopolitical Times (BPT), speculating that the meeting will likely include discussing a mitochondrial replacement technique developed by Shoukhrat Mitalipov at Oregon Health and Science University (OHSU).
Notes the BPT, “mitochondrial replacement is a form of inheritable genetic modification.” This type of gene therapy is the source of much controversy, because it permanently changes the human genome and risks unforeseeable changes in growth and development, and aging.
As late as 2008, all germline modification therapies and enhancements were banned in 83% of the 30 nations making up the OECD (Organization for Economic Cooperation and Development), including the US and UK, reports the Center for Genetics and Society (CGS).
Testifying before the House Foreign Affairs Committee, Subcommittee on Terrorism, Nonproliferation and Trade in 2008, CGS Executive Director Richard Hayes advised:
Most people strongly support therapeutic applications of genetic science, but they also realize that the manipulation of inheritable genetic traits crosses a consequential barrier. In the great majority of instances, couples at risk of passing on a serious genetic disease can ensure that their child is disease-free by means of medically-related trait selection, thus obviating the need for the far more complex and risk-prone intervention that germline modification would entail.
Making humans better, smarter, stronger has long been the goal of eugenicists. Hayes warns:
Germline enhancement has also been seriously proposed as a means of creating people with such novel cognitive, psychological, and behavioral traits that they would constitute a new, ‘post-human’ species, incapable of interbreeding with ‘normal’ humans.
With ooplasmic transfer, the technique injects healthy mitochondrial DNA from a donor into the egg of an infertile woman. Mitochondrial DNA floats outside a cell’s nucleus which contains the regular DNA, and is only inherited from the mother.


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