Huntington Disease

Combination of gene therapy, gene silencing prevents neurodegenerative disease

Published in Gene Therapy Weekly, July 29th, 2004

University of Iowa (UI) researchers have shown for the first time that gene therapy delivered to the brains of living mice can prevent the physical symptoms and neurological damage caused by an inherited neurodegenerative disease that is similar to Huntington disease (HD).

If the therapeutic approach can be extended to humans, it may provide a treatment for a group of incurable, progressive neurological diseases called polyglutamine-repeat diseases, which include HD and several spinocerebellar ataxias. The study, conducted by scientists at the UI Roy J. and Lucille A. Carver College of Medicine and colleagues at the University of Minnesota and the U.S. National...

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Source: Gene Therapy Weekly (2004-07-29)

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