A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy

Recent reports underscore the unparalleled potential of antisense-oligonucleotide (ASO)-based approaches to ameliorate various pathological conditions. However, in vivo studies validating the effectiveness of a short ASO (<10-mer) in the context of a human disease have not been performed. One dis...

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Detalhes bibliográficos
Main Authors: Keil, Jeffrey M, Seo, Joonbae, Howell, Matthew D, Hsu, Walter H, Singh, Ravindra N, DiDonato, Christine J
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4121513/
https://ncbi.nlm.nih.gov/pubmed/25004100
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.23
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