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Cas9 Nickase-Mediated Contraction of CAG/CTG Repeats <i>in Vivo</i> is Accompanied by Improvements in Huntington’s Disease Pathology

2024-02-21

Abstract excerpt

Expanded CAG/CTG repeats cause over 15 different diseases that all remain without a disease-modifying treatment. Because repeat length accounts for most of the variation in disease severity, contracting them presents an attractive therapeutic avenue. Here, we show that the CRISPR-Cas9 nickase targeted to CAG/CTG repeats leads to efficient contractions in Huntington’s disease patient-derived neurons and astrocytes,...

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Literature Corpus work
c4d64a6c-da7c-5582-99a0-784c3d867dd9
DOI
10.1101/2024.02.19.580669
Open publication

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Cas9 Nickase-Mediated Contraction of CAG/CTG Repeats <i>in Vivo</i> is Accompanied by Improvements in Huntington’s Disease PathologyDOI 10.1101/2024.02.19.580669
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