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Small molecule targeting of r(CGG)exp, the RNA causative of fragile X-associated tremor ataxia syndrome (FXTAS). (A) r(CGG)exp sequesters proteins such as DGCR8, Sam68, and hnRNP. (B) Sam68 regulates splicing of SMN2 exon 7. Thus, its sequestration results in increased exon 7 inclusion in the mature SMN2 transcript, contributing to FXTAS pathology. (C) Structures of compounds that bind to r(CGG)exp.
