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

• Главные авторы: Ngoc Lu-Nguyen (Автор), Alberto Malerba (Автор), Linda Popplewell (Автор), Fred Schnell (Автор), Gunnar Hanson (Автор), George Dickson (Автор)
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• Опубликовано: Elsevier, 2017-03-01T00:00:00Z.
• Предметы: article
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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...