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Gene transfer corrects severe muscle defects in mice with Duchenne muscular dystrophy

Gene transfer corrects severe muscle defects in mice with Duchenne muscular dystrophy

Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers have developed a new gene transfer approach that uses an adeno-associated virus vector to deliver a modified dystrophin gene to muscle, restoring muscle strength in a mouse model that closely mimics the severe defects seen in patients.
https://www.sciencedaily.com/releases/2017/07/170727122045.htm

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