Article

Selective targeting of mutant <i>huntingtin</i> intron-1 improves rescue provided by antisense oligonucleotides

2025-07-31

Abstract excerpt

<h4> Abstract </h4> Huntington’s disease (HD) arises from the toxic gain of function caused by a CAG expansion in the coding region of the HTT gene. HD is increasingly appreciated to emerge from multiple pathogenic processes, including somatic instability in mutant HTT’s ( mHTT ) CAG repeat tract, which leads to diverse deleterious consequences. These include the alternative processing of HTT pre-mRNA to gen...

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Literature Corpus work: 46ed43fc-c98d-5a8e-a55e-bf3a7b9a790a
DOI: 10.1101/2025.07.29.665998

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