Transcriptomics

Dataset Information

0

Epigenetic therapy for Friedreich ataxia.


ABSTRACT: We set out to investigate whether a histone deacetylase inhibitor (HDACi) would be effective in an in vitro model for the neurodegenerative disease Friedreich ataxia (FRDA) and to evaluate safety and surrogate markers of efficacy in a phase I clinical trial in patients. In the neuronal cell model, HDACi 109/RG2833 increases FXN mRNA levels and frataxin protein, with concomitant changes in the epigenetic state of the gene. Chromatin signatures indicate that histone H3 lysine 9 is a key residue for gene silencing through methylation and reactivation through acetylation, mediated by the HDACi. Drug treatment in FRDA patients demonstrated increased FXN mRNA and H3 lysine 9 acetylation in peripheral blood mononuclear cells. No safety issues were encountered.

ORGANISM(S): Homo sapiens

PROVIDER: GSE65399 | GEO | 2015/01/29

SECONDARY ACCESSION(S): PRJNA273887

REPOSITORIES: GEO

Dataset's files

Source:
Action DRS
Other
Items per page:
1 - 1 of 1

Similar Datasets

2015-01-29 | E-GEOD-65399 | biostudies-arrayexpress
2008-03-08 | GSE10745 | GEO
2019-12-31 | GSE108200 | GEO
2022-10-04 | GSE200907 | GEO
2023-12-04 | PXD044554 | Pride
2010-07-30 | E-GEOD-22651 | biostudies-arrayexpress
2024-01-29 | GSE244886 | GEO
2010-07-02 | GSE22651 | GEO
2022-11-02 | E-MTAB-11296 | biostudies-arrayexpress
2018-07-11 | PXD006928 | Pride