Journal Article

Gene therapy of muscular dystrophy

Jeffrey S. Chamberlain

in Human Molecular Genetics

Volume 11, issue 20, pages 2355-2362
Published in print October 2002 | ISSN: 0964-6906
Published online October 2002 | e-ISSN: 1460-2083 | DOI: http://dx.doi.org/10.1093/hmg/11.20.2355
Gene therapy of muscular dystrophy

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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.

Journal Article.  7005 words.  Illustrated.

Subjects: Genetics and Genomics

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