NeurosciencebioRxivPreprint
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…·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.