Author

Alys N. Aston

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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. · Science Translational Medici... · 1 citation

#gene editing Open access Oct 2026

Single-AAV Delivery of the SpCas9 Nickase Improves CAG/CTG Repeat Contraction in a Mouse Model of Huntington’s Disease

Delivery of CRISPR-Cas9 nickase in a single AAV improves contraction of CAG repeats in HD and improves the translational potential of AAV-mediated delivery of Cas9.

A. Murillo, Alys N. Aston, M. J. Lelos et al. · bioRxiv · 0 citations

Open access Jul 2026

Targeted sequencing of expanded tandem repeats: Identifying interruptions and errors

CHARLIE (Comprehensive High-throughput Analysis of Repeat Length, Interruptions and Expansions) is developed to identify and position interruptions within expanded repeats.

Aeverie E. R. Heuchan, Ruban Rex Peter Durairaj, Alys N. Aston et al. · bioRxiv · 0 citations