Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease
Doo Eun Choi, Jun Wan Shin, Sophia Zeng, Eun Pyo Hong, Jae-Hyun Jang, Jacob M. Loupe, Vanessa Chantal Wheeler, Hannah E. Stutzman, Ben Kleinstiver, Jong‐Min Lee
The paper develops base editing strategies to convert CAG to CAA in Huntington's disease, reducing CAG repeat expansion and showing therapeutic potential.
eLife · Dec 19, 2023Peer-reviewedGenetics