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...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
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Mary Ann Liebert
2013
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| Online Access: | https://eprints.nottingham.ac.uk/2663/ |