Muscle maturation: implications for gene therapy.
Skeletal muscle is a promising target tissue for gene therapy, for both muscle and non-muscle disorders. A variety of methods have been studied to transfer genes into skeletal muscle, including retroviral, adenoviral and herpes simplex viral vectors. However, various factors impede muscle-based viral gene therapy. Here, we discuss why some viral vectors cannot efficiently transduce mature muscle fibers, and describe some new approaches to overcome this barrier.
van Deutekom
J C
JC
Dept. of Orthopaedic Surgery, Children's Hospital of Pittsburgh, PA 15213, USA.
Hoffman
E P
EP
Huard
J
J
D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects; D005822:Genetic Vectors; D006801:Humans; D051379:Mice; D024510:Muscle Development; D018485:Muscle Fibers, Skeletal / Q000166:cytology* / Q000821:virology; D018482:Muscle, Skeletal / Q000166:cytology / Q000254:growth & development / Q000502:physiology* / Q000821:virology; D009136:Muscular Dystrophies / Q000628:therapy; D009137:Muscular Dystrophy, Animal / Q000628:therapy*; D014780:Viruses / Q000235:genetics