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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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| 主要な著者: | , , , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Blackwell Publishing Ltd
2013
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| 主題: | |
| オンライン･アクセス: | 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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