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XX. Gene Therapy for the Treatment of Genetic Muscle Disease
Jonathan D. Proto, Bing Wang
252 - 261
10.3233/978-1-61499-076-5-252
Biomedical and Health Research
Volume 77: Regenerative Medicine and Cell Therapy
Abstract

With decades of research completed, rAAV-based gene therapies for muscle diseases such as DMD have finally progressed to clinical trials. Multiple approaches, such as gene replacement with mini-dystrophin, gene correction by exon skipping, and reducing chronic inflammation, show promise for the future. Obstacles encountered during human trials have led to novel strategies, including the generation of rAAV pseudo-types, the construction of modified muscle specific promoters, and targeted capsid modifications.

$35.00 / €27.50 / £22.00
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