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Suberoylanilide hydroxamic acid (vorinostat) up-regulates progranulin transcription: rational therapeutic approach to frontotemporal dementia

Cenik, Basar
Sephton, Chantelle F.
Dewey, Colleen M.
Xian, Xunde
Wei, Shuguang
Yu, Kimberley
Niu, Wenze
Coppola, Giovanni
Coughlin, Sarah E.
Lee, Suzee E.
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Abstract

Progranulin (GRN) haploinsufficiency is a frequent cause of familial frontotemporal dementia, a currently untreatable progressive neurodegenerative disease. By chemical library screening, we identified suberoylanilide hydroxamic acid (SAHA), a Food and Drug Administration-approved histone deacetylase inhibitor, as an enhancer of GRN expression. SAHA dose-dependently increased GRN mRNA and protein levels in cultured cells and restored near-normal GRN expression in haploinsufficient cells from human subjects. Although elevation of secreted progranulin levels through a post-transcriptional mechanism has recently been reported, this is, to the best of our knowledge, the first report of a small molecule enhancer of progranulin transcription. SAHA has demonstrated therapeutic potential in other neurodegenerative diseases and thus holds promise as a first generation drug for the prevention and treatment of frontotemporal dementia.

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J Biol Chem. 2011 May 6;286(18):16101-8. Epub 2011 Mar 23. Link to article on publisher's site

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10.1074/jbc.M110.193433
PubMed ID
21454553
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