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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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Detalhes bibliográficos
Main Authors: Yang, Lu, Niu, Hongjing, Gao, Xianjun, Wang, Qingsong, Han, Gang, Cao, Limin, Cai, Chunquan, Weiler, Jan, Yin, Haifang
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2013
Assuntos:
Acesso em linha: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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