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. ... show 9 more
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Authors
Sephton, Chantelle F.
Dewey, Colleen M.
Xian, Xunde
Wei, Shuguang
Yu, Kimberley
Niu, Wenze
Coppola, Giovanni
Coughlin, Sarah E.
Lee, Suzee E.
Dries, Daniel R.
Almeida, Sandra
Geschwind, Daniel H.
Gao, Fen-Biao
Miller, Bruce L.
Farese, Robert V. Jr.
Posner, Bruce A.
Yu, Gang
Herz, Joachim
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UMass Chan Affiliations
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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.
Source
J Biol Chem. 2011 May 6;286(18):16101-8. Epub 2011 Mar 23. Link to article on publisher's site