Article

Droplet digital PCR assay to analyze allele-specific mRNA expression on HTT repeat expansion locus

2025-10-01

Abstract excerpt

<h4>Background</h4> Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by a mutation in the huntingtin gene (HTT), characterized by an expanded CAG trinucleotide repeat. At the time of writing no cure or disease-modifying treatments exist. Currently, the most explored investigational therapeutic strategy targets HTT gene expression, either lowering both alleles (total lowering) or selectively...

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Literature Corpus work: 02ce2709-37b5-57f4-82f5-ede41183ced9
DOI: 10.1101/2025.09.26.25336715

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