Complete restoration of multiple dystrophin isoforms in genetically corrected Duchenne muscular dystrophy patient–derived cardiomyocytes

Abstract

INTRODUCTION Duchenne muscular dystrophy (DMD) is one of the most common and severe inherited neuromuscular disorders, affecting 1 in 3,500 newborn males. DMD is caused by mutations in the dystrophin gene encoding a key structural protein of the dystrophin glycoprotein complex, which connects the contracting cytoskeletal machinery of skeletal and cardiac… (More)

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Cite this paper

@inproceedings{Zatti2014CompleteRO, title={Complete restoration of multiple dystrophin isoforms in genetically corrected Duchenne muscular dystrophy patient–derived cardiomyocytes}, author={Susi Zatti and Sebastian Martewicz and Elena Serena and Narumi Uno and Giovanni G Giobbe and Yasuhiro Kazuki and Mitsuo Oshimura and Nicola Elvassore}, year={2014} }