Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a motor neuron disease, typically resulting from loss-of-function mutations in the survival motor neuron 1 (SMN1) gene. Nusinersen/SPINRAZA, a splice-switching oligonucleotide that modulates SMN2 (a paralog of SMN1) splicing and consequently increases SMN protein lev...

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Publicado no:Sci Rep
Main Authors: Ando, Shiori, Suzuki, Shunya, Okubo, Shoichi, Ohuchi, Kazuki, Takahashi, Kei, Nakamura, Shinsuke, Shimazawa, Masamitsu, Fuji, Koji, Hara, Hideaki
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7562719/
https://ncbi.nlm.nih.gov/pubmed/33060681
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-020-74346-9
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