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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| 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
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| Series: | EMBO Molecular Medicine |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/emmm.201202305 |
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