Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585
Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585
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Publisher
England: Nature Publishing Group
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Language
English
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Publisher
England: Nature Publishing Group
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Contents
Spinal muscular atrophy (SMA) is the leading genetic cause of early childhood death worldwide and no therapy is available today. Many drugs, especially histone deacetylase inhibitors (HDACi), increase SMN levels. As all HDACi tested so far only mildly ameliorate the SMA phenotype or are unsuitable for use in humans, there is still need to identify...
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Full title
Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3658191
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3658191
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ISSN
1018-4813
E-ISSN
1476-5438
DOI
10.1038/ejhg.2012.222
