2′-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping

Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease characterized by dystrophin deficiency from mutations in the dystrophin gene. Antisense oligonucleotide (AO)-mediated exon skipping targets restoration of the dystrophin reading frame to allow production of an internally deleted dys...

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Detalhes bibliográficos
Publicado no:Genes (Basel)
Main Authors: Lee, Tomoko, Awano, Hiroyuki, Yagi, Mariko, Matsumoto, Masaaki, Watanabe, Nobuaki, Goda, Ryoya, Koizumi, Makoto, Takeshima, Yasuhiro, Matsuo, Masafumi
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2017
Assuntos:
Review
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5333056/
https://ncbi.nlm.nih.gov/pubmed/28208626
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/genes8020067
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