Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations

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Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations

With an incidence of 1:3,500 to 5,000 in male children, Duchenne muscular dystrophy (DMD) is an X-linked disorder in which progressive muscle degeneration occurs and affected boys usually die in their twenties or thirties. Cardiac involvement occurs in 90% of patients and heart failure accounts for...

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Bibliographic Details
Main Authors:	Dick, Emily, Kalra, Spandan, Anderson, David, George, Vinoj, Ritso, Morten, Laval, Steven H., Barresi, Rita, Asrtsma-Rus, Annemieke, Lochmueller, Hanns, Denning, Chris
Format:	Article
Published:	Mary Ann Liebert 2013
Online Access:	https://eprints.nottingham.ac.uk/2663/

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