Clinical Development on the Frontier: Gene Therapy for Duchenne Muscular Dystrophy

Asher DR, Thapa K, Dharia SD, et al. Clinical development on the frontier: gene therapy for duchenne muscular dystrophy. Expert Opin Biol Ther 2020;20:263-274   Abstract Introduction: The development of adeno-associated virus (AAV) vectors as safe vehicles for in vivo delivery of therapeutic genes has been a major milestone in the advancement of gene therapy, […]