Make or break for first splice-modulating agents.

作者信息

Mullard Asher

出版信息

Nat Rev Drug Discov. 2013 Nov;12(11):813-5. doi: 10.1038/nrd4151.

DOI:10.1038/nrd4151

PMID:24172317

Abstract

摘要

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Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice.

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