Gene transfer corrects severe muscle defects in mice with Duchenne muscular dystrophy
Thursday, July 27, 2017 - 13:02
in Biology & Nature
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.