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Proposed mode of action of an r(CGG)exp repeat binder that prevents epigenetic silencing of the FMR1 promoter in fragile X syndrome (FXS). (A) Schematic mechanism showing stabilized r(CGG)exp hairpins restrict formation of the RNA:DNA hybrids responsible for epigenetic silencing of FMR1. (B) Schematic mechanism showing binding of a small molecule to the r(CGG)exp hairpin preventing recruitment of polycomb repressive complex 2 (PRC2), a H3K27 methylation enzyme complex.
