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Gene therapy success in mouse model of neurodegenerative disease

By Sharon Kingman
Staff Writer

Monday, April 7, 2014

LONDON – Work on a mouse model representing a rare inherited disease that affects the heart muscle suggests that gene therapy to replace a missing protein may one day be possible in the clinic, for some conditions at least. A team of French researchers used a specific adeno-associated virus that is known to enter heart muscle cells to evaluate how gene therapy could help patients with the neurodegenerative disease, Friedreich’s ataxia. Affecting just one in 50,000 births, Friedreich’s ataxia causes problems with balance and movement. It usually starts in adolescence, with muscle weakness progressing until patients are unable to walk or live independently.

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