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Saponins enhance exon skipping of 2′-O-methyl phosphorothioate oligonucleotide in vitro and in vivo

BACKGROUND: Antisense oligonucleotide (ASO)-mediated exon skipping has been feasible and promising approach for treating Duchenne muscular dystrophy (DMD) in preclinical and clinical trials, but its therapeutic applications remain challenges due to inefficient delivery. METHODS: We investigated a fe...

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Detalles Bibliográficos
Autores principales: Wang, Mingxing, Wu, Bo, Shah, Sapana N, Lu, Peijuan, Lu, Qilong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6217006/
https://www.ncbi.nlm.nih.gov/pubmed/30464402
http://dx.doi.org/10.2147/DDDT.S179008