Ontology highlight
ABSTRACT:
SUBMITTER: Dupont JB
PROVIDER: S-EPMC7000997 | biostudies-literature | 2020 Feb
REPOSITORIES: biostudies-literature
Dupont Jean-Baptiste JB Guo Jianjun J Renaud-Gabardos Edith E Poulard Karine K Latournerie Virginie V Lawlor Michael W MW Grange Robert W RW Gray John T JT Buj-Bello Ana A Childers Martin K MK Mack David L DL
Molecular therapy : the journal of the American Society of Gene Therapy 20191111 2
Multiple clinical trials employing recombinant adeno-associated viral (rAAV) vectors have been initiated for neuromuscular disorders, including Duchenne and limb-girdle muscular dystrophies, spinal muscular atrophy, and recently X-linked myotubular myopathy (XLMTM). Our previous work on a canine model of XLMTM showed that a single rAAV8-cMTM1 systemic infusion corrected structural abnormalities within the muscle and restored contractile function, with affected dogs surviving more than 4 years po ...[more]