Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Antisense-mediated exon skipping is a promising approach for the treatment of Duchenne muscular dystrophy (DMD), a rare life-threatening genetic disease due to dystrophin deficiency. Such an approach can restore the disrupted reading frame of dystrophin pre-mRNA, generating a truncated form of the p...

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Enregistré dans:
Détails bibliographiques
Publié dans:Mol Ther Nucleic Acids
Auteurs principaux: Lu-Nguyen, Ngoc, Malerba, Alberto, Popplewell, Linda, Schnell, Fred, Hanson, Gunnar, Dickson, George
Format: Artigo
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2017
Sujets:
Original Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363451/
https://ncbi.nlm.nih.gov/pubmed/28325281
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2016.11.009
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