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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Hlavní autoři: Dick, Emily, Kalra, Spandan, Anderson, David, George, Vinoj, Ritso, Morten, Laval, Steven H., Barresi, Rita, Aartsma-Rus, Annemieke, Lochmüller, Hanns, Denning, Chris
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2013
Témata:
Original Research Reports
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3787465/
https://ncbi.nlm.nih.gov/pubmed/23829870
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/scd.2013.0135
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