Article

Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington’s disease pathogenesis in mice

2025-06-25

Abstract excerpt

<h4>ABSTRACT</h4> Huntington’s disease (HD) is a progressive neurodegenerative disorder with no approved therapies. Two major molecular drivers—somatic expansion of inherited CAG repeats and toxic mutant HTT (mHTT) variants—lead to neuronal dysfunction. Despite multiple trials, HTT-lowering strategies have not shown meaningful clinical benefit. Using therapeutic divalent siRNAs, we assessed the long-term impact o...

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Literature Corpus work: eafac511-c6ac-5068-b9c8-728bf45cca9b
DOI: 10.1101/2025.06.24.661398

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