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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...
Autores principales: | Yang, Lu, Niu, Hongjing, Gao, Xianjun, Wang, Qingsong, Han, Gang, Cao, Limin, Cai, Chunquan, Weiler, Jan, Yin, Haifang |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2013
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Materias: | |
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 |
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