Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy
Open Access
- 28 September 1995
- journal article
- clinical trial
- Published by Massachusetts Medical Society in New England Journal of Medicine
- Vol. 333 (13) , 832-838
- https://doi.org/10.1056/nejm199509283331303
Abstract
Myoblast transfer has been proposed as a technique to replace dystrophin, the skeletal-muscle protein that is deficient in Duchenne's muscular dystrophy. Donor myoblasts injected into muscles of affected patients can fuse with host muscle fibers, thus contributing their nuclei, which are potentially capable of replacing deficient gene products. Previous controlled trials involving a single transfer of myoblasts have been unsuccessful.Keywords
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