The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy
Niclas E. Bengtsson, Hichem Tasfaout, Jeffrey S. Chamberlain
Topics & Concepts
Duchenne muscular dystrophyGenetic enhancementGeneMuscular dystrophyMedicineBiologyVirologyGeneticsVirus-based gene therapy researchMuscle Physiology and DisordersRNA Interference and Gene Delivery