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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Wu, Bo, Lu, Peijuan, Cloer, Caryn, Shaban, Mona, Grewal, Snimar, Milazi, Stephanie, Shah, Sapana N., Moulton, Hong M., Lu, Qi Long
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society for Investigative Pathology 2012
Pynciau:
Regular Article
Mynediad Ar-lein: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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