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Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45–55 Skipping Accompanied by Rescue of Dystrophin Expression

Antisense oligonucleotide-mediated exon skipping is a promising therapeutic approach for the treatment of various genetic diseases and a therapy which has gained significant traction in recent years following FDA approval of new antisense-based drugs. Exon skipping for Duchenne muscular dystrophy (D...

詳細記述

保存先:
書誌詳細
出版年:Methods Mol Biol
主要な著者: Lee, Joshua J. A., Saito, Takashi, Duddy, William, Takeda, Shin’ichi, Yokota, Toshifumi
フォーマット: Artigo
言語:Inglês
出版事項: 2018
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6578579/
https://ncbi.nlm.nih.gov/pubmed/30171539
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/978-1-4939-8651-4_8
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