Retroviral vectors for gene therapy of Duchenne muscular dystrophy

Citation
A. Fassati et N. Bresolin, Retroviral vectors for gene therapy of Duchenne muscular dystrophy, NEUROL SCI, 21(5), 2000, pp. S925-S927
Citations number
5
Categorie Soggetti
Neurology
Journal title
NEUROLOGICAL SCIENCES
ISSN journal
15901874 → ACNP
Volume
21
Issue
5
Year of publication
2000
Supplement
S
Pages
S925 - S927
Database
ISI
SICI code
1590-1874(2000)21:5<S925:RVFGTO>2.0.ZU;2-E
Abstract
The aim of this review is to summarize the state of art of retroviral vecto rs for gene therapy of Duchenne muscular dystrophy (DMD). Actual knowledge on this matter indicates that retroviral vectors are able to transduce musc le satellite cells ill 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 normaliz ation process of DMD carriers and offers hope for the development of long-t erm gene therapy of this disease.