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Article

Selective targeting of mutant huntingtin intron 1 improves rescue provided by antisense oligonucleotides in Huntington's disease mice.

Science translational medicine - 18 Mar 2026

Bragg Robert M, Landles Christian, Smith Edward J, Osborne Georgina F, Mathews Ella W, Cantle Jeffrey P, Bates Gillian P, Carroll Jeffrey B

Abstract excerpt

Huntington's disease (HD) arises from the toxic gain of function caused by a CAG expansion in the coding region of the huntingtin (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 generate the...

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Identifiers

PMID: 41849580
DOI: 10.1126/scitranslmed.adv0702

Genes

HTT

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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Selective targeting of mutant huntingtin intron 1 improves rescue provided by antisense oligonucleotides in Huntington's disease mice.PMID 41849580Read ↗

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