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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Päätekijät: Yang, Lu, Niu, Hongjing, Gao, Xianjun, Wang, Qingsong, Han, Gang, Cao, Limin, Cai, Chunquan, Weiler, Jan, Yin, Haifang
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Public Library of Science 2013
Aiheet:
Research Article
Linkit: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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