Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells
Abstract
The protein dysferlin is abundantly expressed in skeletal and cardiac muscles, where its main function is membrane repair. Mutations in the dysferlin gene are involved in two autosomal recessive muscular dystrophies: Miyoshi myopathy and limb‐girdle muscular dystrophy type 2B. Development of effective therapies remains a great challenge. Strategies to repair the dysferlin gene by skipping mutated exons, using antisense oligonucleotides ( AON s),...
Paper Details
Title
Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells
Published Date
Oct 8, 2013
Journal
Volume
280
Issue
23
Pages
6045 - 6060
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