Gene therapy: a strategy for the treatment of inherited muscle diseases?
The emergence of new vectors of viral origin (recombinant adeno-associated viruses, second and third generation adenoviruses) and a new potential source of cells for transplantation (muscle-derived stem cells) are broadening the panel of therapeutic options for myopathies. Although the perfect gene-transfer method(s) have not yet been found, recent findings will certainly constitute a strong knowledge base for future clinical trials.
Moisset
P A
PA
Human Genetics Unit, CHUL Research Center, Laval University, Ste-Foy, Quebec, Canada.
Tremblay
J P
JP
D000818:Animals; D015316:Genetic Therapy; D006801:Humans; D018482:Muscle, Skeletal / Q000502:physiology; D009135:Muscular Diseases / Q000235:genetics* / Q000628:therapy*