Precise CAG repeat contraction in a Huntington’s Disease mouse model is enabled by gene editing with SpCas9-NG

Abstract

Seiya Oura and Taichi Noda et al. overcome the challenge of gene editing in CAG repeats, such as those causing Huntington’s Disease, using their recently developed SpCas9-NG variant. They demonstrate that SpCas9-NG can precisely edit and contract the CAG repeat tracks in a Huntington’s Disease mouse model, opening new avenues for research in this disease.