Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic mdx Mice by Peptide-Conjugated Morpholino

Exon skipping is capable of correcting frameshift and nonsense mutations in Duchenne muscular dystrophy. Phase 2 clinical trials in the United Kingdom and the Netherlands have reported induction of dystrophin expression in muscle of Duchenne muscular dystrophy patients by systemic administration of...

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Autori principali: Wu, Bo, Lu, Peijuan, Cloer, Caryn, Shaban, Mona, Grewal, Snimar, Milazi, Stephanie, Shah, Sapana N., Moulton, Hong M., Lu, Qi Long
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Investigative Pathology 2012
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Regular Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3409432/
https://ncbi.nlm.nih.gov/pubmed/22683468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajpath.2012.04.006
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