Reprogrammed Müller Glia Restore Vision in Mice
Two gene injections reprogram Müller glia into rod photoreceptors in the retinas of blind mice and partially restore the animals’ vision, researchers report today (August
Two gene injections reprogram Müller glia into rod photoreceptors in the retinas of blind mice and partially restore the animals’ vision, researchers report today (August
The National Institutes of Health (NIH) and the Food and Drug Administration (FDA) have played key roles in the emergence of safe and effective human
Researchers at Caltech have designed a noninvasive method to control specific neural circuits in the mouse brain. The technique, published earlier this week (July 9)
Due to advances in rare-disease research and individualized cell and gene therapies, there has been a recent crop of treatments approved by regulatory agencies not
The body’s own immune system could thwart some efforts to develop gene therapies based on the trendy genome-editing tool called CRISPR-Cas9, according to a study
Researchers have successfully treated animals with a mouse version of type 1 diabetes by inducing pancreatic cells that don’t normally produce insulin to manufacture the
FDA approved Spark Therapeutics’ gene therapy Luxturna™ (voretigene neparvovec-rzyl) for treating children and adults with the rare inherited blindness disorder biallelic RPE65 mutation-associated retinal dystrophy.
arly in 2016, a woman went to a clinic in Dallas to have a genetically modified adeno-associated virus injected into her eye. The woman was
Researchers have edited the human genome before, but always in cells outside the body. Now, biotech company Sangamo Therapeutics is recruiting participants for clinical trials
In a first, a man has received a therapy aimed at editing the genes inside his body. The Associated Press reports that 44-year-old Brian Madeux,
Thanks to an international team of scientists and doctors, a seven-year-old Syrian refugee who lost most of his outer skin to a life-threatening genetic disease
Researchers have edited the human genome before, but always in cells outside the body. Now, biotech company Sangamo Therapeutics is recruiting participants for clinical trials
Advancing age predisposes us to a number of neurodegenerative diseases, yet the underlying mechanisms are poorly understood. With some 70 million individuals affected, glaucoma is
It is not easy being a cancer cell, so such cells may need help from factors other than oncogenes that contribute to the cancer cell
With over 10 million active cases in 2015 and close to 2 million deaths, tuberculosis (TB) remains a leading cause of death worldwide. Moreover, the
Last year, Elizabeth Parrish, the CEO of Seattle-based biotech firm BioViva, hopped a plane to Colombia, where she received multiple injections of two experimental gene
Preserving the distinction between research purposes and clinical applications, the Human Fertilisation and Embryology Authority (HFEA), a U.K. regulatory body, has approved the use of
A piece of noncoding DNA helps prevents cells from turning cancerous, and does so via two mechanisms. The noncoding DNA, which is called GNG12-ASI, acts
The hottest technique in gene editing, called CRISPR, could soon be used to study human embryos. On 14 January, a regulatory committee in the United
Adenovectors are hot again. Two decades after the initial flurry of excitement about gene-based medicine, researchers and the media are once again enthusiastic about the
If a Cas9 nuclease variant could be engineered that was less grabby, it might loosen its grip on DNA sequences throughout the genome except those
Scientists in Korea have injected human embryonic stem cell (hESC)-derived retinal support cells into the eyes of four men with macular degeneration, according to a
Patients with Leber’s congenital amaurosis (LCA), a genetic blindness disorder caused by a mutation in the retinal pigment epithelium 65 (RPE65) gene that encodes a
A two-armed molecule has been developed that effectively locks HIV in a kind of miniature full nelson, preventing the virus from wrestling its way into
Scientists at the Harvard Stem Cell Institute at Massachusetts General and Boston Children’s hospitals say that a novel gene editing method has been used to
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