The human population carries around more deadly genetic diseases than would be expected based on a simple comparison of mutation rates and deaths of affected individuals. Carlos Eduardo Guerra Amorim of Stony Brook University ...
Sep 28, 2017
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The first known identification of two genes responsible for hypoplastic left heart syndrome (HLHS), a severe congenital heart defect, has been reported by researchers at the University of Pittsburgh School of Medicine. The ...
May 22, 2017
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(HealthDay)—The U.S. Food and Drug Administration says it has expanded approval for the cystic fibrosis drug Kalydeco (ivacaftor) to include 33 mutations of the disease, up from the previous 10 mutations.
May 18, 2017
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A group of researchers has found that three siblings born with cleft lip and palate share a common gene mutation associated with the birth defect.
Mar 21, 2017
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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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