Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease. ...
Jan 4, 2022
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Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A. This pharmacology study, performed in a mouse model and designed to support the initiation of ...
Dec 16, 2021
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This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional ...
Nov 30, 2021
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Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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A novel computational platform developed by researchers from the University of Pittsburgh School of Medicine identifies top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency ...
Oct 19, 2021
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A devastating genetic disease called CDKL5 deficiency disorder (CDD), which strikes in early childhood, may be significantly treatable even in adulthood, a new study from the Perelman School of Medicine at the University ...
Oct 15, 2021
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Autosomal dominant polycystic kidney disease (ADPKD), a genetic disorder, causes fluid-filled cysts to develop on the kidneys, which can impair their function. As part of the growth of cysts, the kidneys develop inflammation ...
Oct 13, 2021
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In the last few years, an alternative for these cases has emerged: a cell therapy known as CAR-T that collects the T-lymphocytes of these patients, modifies them through genetic engineering in the laboratory and administer ...
Oct 4, 2021
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The NHS has very recently approved a new cholesterol-lowering jab which will be offered to 300,000 people over the next three years.
Sep 10, 2021
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Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
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