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In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient

Antisense oligonucleotide induced exon skipping emerges as a promising therapeutic strategy for patients suffering from a devastating muscle disorder Duchenne muscular dystrophy (DMD). Systemic administration of antisense phosphorodiamidate morpholino oligomers (PMOs) targeting exons 6 and 8 in dyst...

詳細記述

保存先:
書誌詳細
出版年:Methods Mol Biol
主要な著者: Nakamura, Akinori, Aoki, Yoshitsugu, Tsoumpra, Maria, Yokota, Toshifumi, Takeda, Shin’ichi
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/PMC6557157/
https://ncbi.nlm.nih.gov/pubmed/30171540
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/978-1-4939-8651-4_9
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