Nerve axons serve as the wiring of the nervous system, sending electrical signals that control movement and sense of touch. When axons are damaged, whether by injury or as a side effect of certain drugs, a program is triggered ...
Jan 17, 2019
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Medicine is at a turning point, on the cusp of major change as disruptive technologies such as gene, RNA, and cell therapies enable scientists to approach diseases in new ways. The swiftness of this change is being driven ...
Jan 10, 2019
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Mice with heart failure that were treated with AAV8-based gene therapy to deliver the protein urocortin 3 (UCn3) had increased blood levels of UCn3 over a 5-week period and improved heart function. The mice received a single ...
Nov 14, 2018
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A combinatorial gene therapy approach- one designed to inhibit inflammation and one targeting protection against cartilage degeneration—was shown to preserve articular cartilage better than each approach alone in animal ...
Nov 13, 2018
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Researchers have taken advantage of the benefits of DNA immunization over traditional protein-based immunization to elicit monoclonal antibodies (mAbs) against challenging targets in three species – mouse, rabbit, and human ...
Oct 22, 2018
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Recently, the U.S. National Institutes of Health (NIH) and Food and Drug Administration (FDA) called for the eliminating involvement of the Recombinant DNA Advisory Committee (RAC) in human gene therapy experiments, marking ...
Sep 19, 2018
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Back in 2009, a group of Israeli researchers identified a herd of Awassi sheep suffering from "day blindness". As its name implies, these sheep were blind during the day (in bright light) but could see at night, in low-light ...
Sep 17, 2018
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Gene therapy using an optimized adeno-associated virus (AAV) to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks ...
Aug 20, 2018
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A study of a large animal model of achromatopsia caused by a mutation in the CNGA3 gene that was treated with a single injection of CNGA3 gene therapy delivered using an AAV5 vector revealed findings reported long-term follow-up ...
Aug 15, 2018
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Researchers at UMass Medical School have developed a genome-editing strategy to correct disease-causing DNA mutations in mouse models of human genetic diseases, according to research published in the Aug. 18 edition of Nature ...
Aug 14, 2018
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