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Somatic CRISPR editing of <i>Msh3</i> mitigates Huntington’s disease pathology in mice

2026-06-10

Abstract excerpt

<h4>ABSTRACT</h4> Huntington’s disease (HD) is a fatal, dominantly inherited neurodegenerative disorder caused by a CAG repeat expansion in Huntingtin ( HTT ) exon 1. Further progressive CAG repeat expansion occurs in somatic cells, particularly in neurons, and drives the timing of clinical onset. Therefore, therapeutic strategies to slow somatic expansion are predicted to be disease-modifying. Somatic CAG expan...

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Literature Corpus work
8943390e-b71a-5496-a09d-a9591d8c5d64
DOI
10.64898/2026.06.08.730940
Open publication

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Somatic CRISPR editing of <i>Msh3</i> mitigates Huntington’s disease pathology in miceDOI 10.64898/2026.06.08.730940
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