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Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models

18 Aug 2024

Abstract excerpt

Huntington's disease (HD) is a fatal neurodegenerative disease caused by a trinucleotide repeat expansion in exon 1 of the huntingtin gene (HTT) that results in toxic gain of function and cell death. Despite its monogenic cause, the pathogenesis of HD is highly complex, and increasing evidence indicates that, in addition to the full-length (FL) mutant HTT protein, the expanded exon 1 HTT (HTTexon1) protein that...

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PMID: 39155061
DOI: 10.1093/brain/awae266

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