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Article

Small Molecules Targeting H3K9 Methylation Prevent Silencing of Reactivated FMR1 Alleles in Fragile X Syndrome Patient Derived Cells.

Genes - 27 Mar 2020

Kumari Daman, Sciascia Nicholas, Usdin Karen

Abstract excerpt

In fragile X syndrome (FXS), expansion of a CGG repeat tract in the 5'-untranslated region of the FMR1 gene to >200 repeats causes transcriptional silencing by inducing heterochromatin formation. Understanding the mechanism of FMR1 silencing is important as gene reactivation is a potential treatment approach for FXS. To date, only the DNA demethylating drug 5-azadeoxycytidine (AZA) has proved effective at gene...

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Identifiers

PMID: 32230785
DOI: 10.3390/genes11040356
PMCID: PMC7230530

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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