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Author

Meghan Larin

1 paper indexed here

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Open access Oct 2026

Cas9 nickase-mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington's disease mouse model.

Expanded CAG/CTG repeats cause more than 15 different diseases that all remain without a disease-modifying treatment. Because repeat length accounts for most of the variation in disease severity, contracting CAG/CTG repeats presents an attractive therapeutic avenue. Here, we show that the CRISPR-Cas9 nickase targeted t...

A. Murillo, M. Alpaugh, Ruban Peter-Durairaj et al. · 1 citation

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