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Internal deletion compromises the stability of dystrophin

Duchenne muscular dystrophy (DMD) is a deadly and common childhood disease caused by mutations that disrupt dystrophin protein expression. Several miniaturized dystrophin/utrophin constructs are utilized for gene therapy, and while these constructs have shown promise in mouse models, the functional...

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Hlavní autoři: Henderson, Davin M., Belanto, Joseph J., Li, Bin, Heun-Johnson, Hanke, Ervasti, James M.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2011
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3131041/
https://ncbi.nlm.nih.gov/pubmed/21558423
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddr199
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