Micro-dystrophin: A Small Gene With Big Promise
Micro-dystrophin: A Small Gene With Big Promise https://pediatricsnationwide.org/wp-content/uploads/2023/04/Spring-23-Cover-Final-crop-1024x632.jpg 1024 632 Abbie Miller https://pediatricsnationwide.org/wp-content/uploads/2023/05/051023BT016-Abbie-Crop.jpg
SRP-9001 for Duchenne muscular dystrophy supplies a functional dystrophin gene via AAVrh74 gene therapy. In 1969, Jerry Mendell, MD, was working at the National Institute of Neurological Disorders and Stroke (NINDS) when he saw his first patient with Duchenne muscular dystrophy (DMD). DMD, a severe form of muscular dystrophy caused by a mutation in the…