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

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

Abstract 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 f...

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Bibliographic Details
Main Authors: Jonathan J. Cherry, Erkan Y. Osman, Matthew C. Evans, Sungwoon Choi, Xuechao Xing, Gregory D. Cuny, Marcie A. Glicksman, Christian L. Lorson, Elliot J. Androphy
Format: Article
Language:English
Published: Springer Nature 2013-06-01
Series:EMBO Molecular Medicine
Subjects:
drug discovery
SMA
SMN
SMN2
spinal muscular atrophy
Online Access:https://doi.org/10.1002/emmm.201202305
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https://doi.org/10.1002/emmm.201202305

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