Problems and potential for gene therapy in Duchenne muscular dystrophy.

doi:10.1016/s0960-8966(97)00056-4

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Article

Problems and potential for gene therapy in Duchenne muscular dystrophy.

Neuromuscular disorders : NMD - 1 Jul 1997

Kakulas B A

Abstract excerpt

Hopes ran high that a cure for Duchenne muscular dystrophy (DMD) would quickly follow the discovery of dystrophin by Lou Kunkel and his group in the 1980's. Myoblast transplantation, the favoured method of gene 'complementation', unfortunately did not progress beyond the experimental stage. A mor...

Topics

Dystrophin
Genetic Therapy
Humans
Microscopy, Electron
Muscular Dystrophies
Mutation

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