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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Detalles Bibliográficos
Autores principales: Yang, Lu, Niu, Hongjing, Gao, Xianjun, Wang, Qingsong, Han, Gang, Cao, Limin, Cai, Chunquan, Weiler, Jan, Yin, Haifang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2013
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3637291/
https://www.ncbi.nlm.nih.gov/pubmed/23658612
http://dx.doi.org/10.1371/journal.pone.0061584

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3637291/
https://www.ncbi.nlm.nih.gov/pubmed/23658612
http://dx.doi.org/10.1371/journal.pone.0061584

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