Challenges for gene therapy for muscular dystrophy.
Gene therapy for muscular dystrophy represents a promising avenue of pursuit for a disease with a limited repertoire of treatment. Recent successes in the research arena using adeno-associated viral vectors should accelerate the movement of gene-based therapeutics for muscle disorders into the clinic. Nevertheless, significant challenges remain before gene therapy can deliver on the promises avowed by early pioneers of the field. This review examines recent progress and the hurdles remaining to achieve gene-based treatment therapies for muscular dystrophy.
Center for Gene Therapy, Columbus Children's Research Institute, Columbus, OH 43205, USA. mendellj@ccri.net
Jerry R
JR
Mendell
K Reed
KR
Clark
D002986:Clinical Trials as Topic; D003599:Cytoskeleton / Q000378:metabolism; D000229:Dependovirus / Q000235:genetics / Q000378:metabolism; D016189:Dystrophin / Q000235:genetics / Q000378:metabolism; D004705:Endocytosis / Q000502:physiology; D015316:Genetic Therapy / Q000009:adverse effects; D006801:Humans; D007107:Immune System / Q000502:physiology; D009136:Muscular Dystrophies / Q000235:genetics / Q000628:therapy*; D009154:Mutation; D014161:Transduction, Genetic
D016189:Dystrophin