Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model

Spinal muscular atrophy (SMA) is a motor neuron disease. Nusinersen, a splice-switching antisense oligonucleotide (ASO), was the first approved drug to treat SMA. Based on prior preclinical studies, both 2′-O-methoxyethyl (MOE) with a phosphorothioate backbone and morpholino with a phosphorodiamidat...

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Detalles Bibliográficos
Autores principales: Sheng, Lei, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R, Hua, Yimin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7102994/
https://www.ncbi.nlm.nih.gov/pubmed/32103257
http://dx.doi.org/10.1093/nar/gkaa126

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7102994/
https://www.ncbi.nlm.nih.gov/pubmed/32103257
http://dx.doi.org/10.1093/nar/gkaa126

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