Effective Exon Skipping and Dystrophin Restoration by 2′-O-Methoxyethyl Antisense Oligonucleotide in Dystrophin-Deficient Mice

Antisense oligonucleotide (AO)–mediated exon-skipping therapy is one of the most promising therapeutic strategies for Duchenne Muscular Dystrophy (DMD) and several AO chemistries have been rigorously investigated. In this report, we focused on the effect of 2′-O-methoxyethyl oligonucleotides (MOE) o...

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Bibliografiske detaljer
Main Authors: Yang, Lu, Niu, Hongjing, Gao, Xianjun, Wang, Qingsong, Han, Gang, Cao, Limin, Cai, Chunquan, Weiler, Jan, Yin, Haifang
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2013
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3637291/
https://ncbi.nlm.nih.gov/pubmed/23658612
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0061584
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