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Human Molecular Genetics, 2002, Vol. 11, No. 20 2355-2362
© 2002 Oxford University Press

Gene therapy of muscular dystrophy

Jeffrey S. Chamberlain*

Department of Neurology, K243b HSB Box 357720, University of Washington School of Medicine, Seattle, WA, 98195-7720, USA

Received August 2, 2002; Accepted August 8, 2002

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.

* To whom correspondence should be addressed. Tel: +1 2066166645; Fax: +1 2066168272; Email: jsc5{at}u.washington.edu


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