Cas9 Nickase-Mediated Contraction of CAG/CTG Repeats in Vivo is Accompanied by Improvements in Huntington's Disease Pathology
Alvaro Murillo, Melanie Alpaugh, Ruban Rex Peter Durairaj, Emma L. Randall, Meghan Larin, Laura Heraty, Alys N Aston, Alysha S Taylor, Alex Mas Monteys, Nina Stöberl, Aeverie E. R. Heuchan, Pascale C. Aeschlimann, Kyle Fears, Christian Landles, Georgina F Osborne, , Vincent Dion
The paper demonstrates that CRISPR-Cas9 nickase can contract CAG/CTG repeats in patient-derived cells and improve molecular and behavioral phenotypes in Huntington's disease.
bioRxiv · Feb 19, 2024 · PreprintNeuroscience