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Article

Epigenetic modulation through BET bromodomain inhibitors as a novel therapeutic strategy for progranulin-deficient frontotemporal dementia.

Scientific reports - 20 Apr 2024

Rosenthal Zachary C, Fass Daniel M, Payne N Connor, She Angela, Patnaik Debasis, Hennig Krista M, Tesla Rachel, Werthmann Gordon C, Guhl Charlotte, Reis Surya A, Wang Xiaoyu, Chen Yueting, Placzek Michael, Williams Noelle S, Hooker Jacob, Herz Joachim, Mazitschek Ralph, Haggarty Stephen J

Abstract excerpt

Frontotemporal dementia (FTD) is a debilitating neurodegenerative disorder with currently no disease-modifying treatment options available. Mutations in GRN are one of the most common genetic causes of FTD, near ubiquitously resulting in progranulin (PGRN) haploinsufficiency. Small molecules that can restore PGRN protein to healthy levels in individuals bearing a heterozygous GRN mutation may thus have...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 38643236
DOI: 10.1038/s41598-024-59110-7
PMCID: PMC11032351

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