New research sheds light on common pathogenic mechanisms shared by Huntington's disease (HD) and HD-like disorders. The study, published by Cell Press in the May 12, 2011, issue of the journal Neuron, uses a new transgenic ...
May 11, 2011
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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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Gene therapy was successfully used to overcome the cardiac effects of Freidreich's ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy.
Sep 18, 2020
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An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe disease ...
Aug 13, 2019
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Dysfunctional neurons in the hippocampus of adult female mice modeling dementia can be repaired and reconnected to distant parts of the brain, reports a new study published in JNeurosci. The similarity between the mouse model ...
May 27, 2019
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When Katie O'Nell's high school biology teacher showed a NOVA video on epigenetics after the AP exam, he was mostly trying to fill time. But for O'Nell, the video sparked a whole new area of curiosity.
Apr 19, 2019
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A new study has shown that a single injection of a novel adeno-associated vector (AAV)-based therapy can result in improved enzyme activ-ity and glycogen clearance as well as prolonged survival in a mouse model of Pompe dis-ease. ...
Aug 13, 2018
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Natural killer (NK) cells which belong in innate immunity, play an important role for defense against invading pathogens and microbes. NK cells make up around 5-20% of the white blood cells circulating in the bloodstream ...
Mar 27, 2017
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Genetic ablation of P2RX7 can improve muscle function and partially correct cognitive impairment and bone loss in a mouse model of Duchenne muscular dystrophy (DMD), according to a study published this week in PLOS Medicine. ...
Oct 13, 2015
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Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and ...
Aug 31, 2015
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