Article

Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidence

2025-04-28

Abstract excerpt

Huntington disease (HD) is caused by inherited CAG expansions, which continue expanding somatically in affected brain regions to hasten disease onset and progression. Therapeutically diminishing somatic expansions is expected to be clinically beneficial. However, it is not known if interventionally modifying somatic CAG expansions will actually modify in vivo clinically-relevant phenotypes, what the therapeutic w...

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Literature Corpus work: d4959004-7522-5bd9-82d1-b9d03678b2c0
DOI: 10.1101/2025.04.25.650652

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