Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy. A team of researchers ...
Mar 21, 2017
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Researchers have started a new gene therapy clinical trial to treat X-linked retinitis pigmentosa (XLRP), the most common cause of blindness in young people.
Mar 20, 2017
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Scientists have developed a new approach to repair a defective gene in blood-forming stem cells from patients with a rare genetic immunodeficiency disorder called X-linked chronic granulomatous disease (X-CGD). After transplant ...
Jan 11, 2017
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A defect in a gene that produces dopamine in the brain appears to accelerate the onset of Parkinson's disease, according to new research from Iowa State University. The effect is particularly dramatic for people under age ...
Nov 28, 2016
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A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
Nov 4, 2016
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For the first time, National Institutes of Health (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). ...
Oct 26, 2016
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Duchenne is the most common and severe form of muscular dystrophy. Because of this genetic disease, one out of every 3,500 children spends their 12th birthday in a wheelchair. This disorder progressively leads to general ...
Oct 19, 2016
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An experimental model uses genetics-guided biomechanics and patient-derived stem cells to predict what type of inherited heart defect a child will develop, according to authors of a new study in the journal Cell.
May 19, 2016
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Many diseases, such as cancer, diabetes, and schizophrenia, tend to be passed down through families. After researchers sequenced the human genome about 15 years ago, they had high hopes that this trove of information would ...
May 11, 2016
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(Medical Xpress)—A team of researchers from several instantiations in the U.S. has found a connection between two defective genes in humans and the messages that are sent from a type of good bacteria and Irritable Bowel ...
