Engineered CRISPR-Base Editors as a Permanent Treatment for Familial Dysautonomia
Shuqi Yun, Anil K. Chekuri, Jennifer Art, Krishnakanth Kondabolu, Samikshya Pokharel, Paolo Pigini, Sydnee Dymock, David Rufino-Ramos, Susan A. Slaugenhaupt, Nadja Zeltner, Benjamin P. Kleinstiver, Elisabetta Morini, Christiano R. R. Alves
This paper develops a CRISPR base editing strategy to correct the FD-causing mutation in human cells and mice, achieving up to 70% on-target correction and functional restoration.
bioRxiv · Nov 27, 2024 · PreprintBioengineering