Retroviral vectors for gene therapy of Duchenne muscular dystrophy.
The aim of this review is to summarize the state of art of retroviral vectors for gene therapy of Duchenne muscular dystrophy (DMD). Actual knowledge on this matter indicates that retroviral vectors are able to transduce muscle satellite cells in vivo and that these cells can participate in muscle repair processes, even if the efficiency of transduction of satellite cells remains the limiting factor. Such a process is reminiscent of the normalization process of DMD carriers and offers hope for the development of long-term gene therapy of this disease.
Fassati
A
A
Wohl Virion Centre, Windeyer Institute, University College London, 46 Cleveland Street, London W1P 6DB, UK.
Bresolin
N
N
D000818:Animals; D016189:Dystrophin / Q000235:genetics / Q000378:metabolism; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics / Q000627:therapeutic use*; D006801:Humans; D024510:Muscle Development; D018482:Muscle, Skeletal / Q000166:cytology / Q000254:growth & development / Q000378:metabolism; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D012190:Retroviridae / Q000235:genetics*; D014161:Transduction, Genetic
D016189:Dystrophin