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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Autors principals:	Lu Yang, Hongjing Niu, Xianjun Gao, Qingsong Wang, Gang Han, Limin Cao, Chunquan Cai, Jan Weiler, Haifang Yin
Format:	Artigo
Idioma:	Inglês
Publicat:	Public Library of Science (PLoS) 2013-01-01
Col·lecció:	PLoS ONE
Accés en línia:	http://europepmc.org/articles/PMC3637291?pdf=render
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