Article

Lowering the <i>HTT1a</i> transcript as an effective therapy for Huntington’s disease

2025-06-11

Abstract excerpt

Lowering the levels of HTT transcripts has been a major focus of therapeutic development for Huntington’s disease (HD), but which transcript should be lowered? HD is caused by a CAG repeat expansion in exon 1 of the HTT gene, and the rate of somatic expansion of this CAG repeat throughout life is now known to drive the age of onset and rate of disease progression. As the CAG repeat expands, the extent to which t...

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Literature Corpus work: b35f990b-891e-5643-b40f-2276c15b20a3
DOI: 10.1101/2025.06.10.658804

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