Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds

Spinal muscular atrophy (SMA) is a neurodegenerative disease that causes progressive muscle weakness, which primarily targets proximal muscles. About 95% of SMA cases are caused by the loss of both copies of the SMN1 gene. SMN2 is a nearly identical copy of SMN1, which expresses much less functional...

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Autors principals: Cherry, Jonathan J, Osman, Erkan Y, Evans, Matthew C, Choi, Sungwoon, Xing, Xuechao, Cuny, Gregory D, Glicksman, Marcie A, Lorson, Christian L, Androphy, Elliot J
Format: Artigo
Idioma:Inglês
Publicat: Blackwell Publishing Ltd 2013
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Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3721476/
https://ncbi.nlm.nih.gov/pubmed/23740718
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201202305
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