Splice modulators target PMS1 to reduce somatic expansion of the Huntington’s disease-associated CAG repeat
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DOI: 10.1038/s41467-024-47485-0
Abstract: Huntington’s disease (HD) is a dominant neurological disorder caused by an expanded HTT exon 1 CAG repeat that lengthens huntingtin’s polyglutamine tract. Lowering mutant huntingtin has been proposed for treating HD, but genetic modifiers implicate… read more here.
Keywords: repeat; splice modulators; cag repeat; huntington disease ... See more keywords