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

Thursday, July 27, 2017

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.