Article
Challenges for gene therapy for muscular dystrophy.
Current neurology and neuroscience reports - 1 Jan 2006
Mendell Jerry R, Clark K Reed
Abstract excerpt
Gene therapy for muscular dystrophy represents a promising avenue of pursuit for a disease with a limited repertoire of treatment. Recent successes in the research arena using adeno-associated viral vectors should accelerate the movement of gene-based therapeutics for muscle disorders into the clinic. Nevertheless, significant challenges remain before gene therapy can deliver on the promises avowed by early...
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