Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease
Katherine Tan, Daniela Del Bosque Siller, Alisha Y. Xiong, Anna X.A. Wang, Tristan X. McCallister, Sreya Mummadi, Luke A. St John, Tae Kyu Lee, Annika Carrillo, Daniel G. Renshaw, Richard H. Zhou, Colin K.W. Lim, Jack He, Christopher J. Fields, Michael R. Hayden, Thomas Gaj
This study develops a CRISPR-Cas9 system targeting all HTT alleles in HD mouse models, reducing mutant HTT and improving disease symptoms.
Molecular Therapy · Apr 24, 2026Peer-reviewedBioengineering