Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug‐like...
Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug‐like compounds
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Publisher
London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Contents
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 SMN protein.
SMN2
is una...
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Full title
Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug‐like compounds
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TN_cdi_doaj_primary_oai_doaj_org_article_d87b1837bfc644f1a80827699a5c93bc
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_d87b1837bfc644f1a80827699a5c93bc
Other Identifiers
ISSN
1757-4676
E-ISSN
1757-4684
DOI
10.1002/emmm.201202305
