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Review
. 2002 Oct 1;11(20):2355-62.
doi: 10.1093/hmg/11.20.2355.

Gene therapy of muscular dystrophy

Jeffrey S Chamberlain  1
Affiliations
Review

Gene therapy of muscular dystrophy

Jeffrey S Chamberlain. Hum Mol Genet. .

Abstract

Development of gene therapy for the muscular dystrophies represents a daunting challenge requiring significant advances in our knowledge of the defective genes, muscle promoters, viral vectors, immune system surveillance and methods for systemic delivery of vectors. However, tremendous progress has been made in developing improved viral vectors and avoiding immune reactions against gene transfer. This review summarizes recent progress and highlights problems that must be solved before an effective treatment is available.

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