Two therapeutic CRISPR/Cas9 gene editing approaches revert FTD/ALS cellular pathology caused by a C9orf72 repeat expansion mutation in patient derived cells
Maria Sckaff, Kamaljot Gill, Aradhana Sachdev, Alisha M. Birk, Olubankole Aladesuyi Arogundade, HL Watry, Kathleen C. Keough, Y-C Tsai, Janet S. Ziegle, Bruce R. Conklin, Claire Dudley Clelland
This study evaluates three CRISPR/Cas9 approaches to correct C9orf72 repeat expansion in patient-derived neurons, finding that repeat and mutant allele excision fully eliminate dipeptide repeats.
bioRxiv · May 21, 2022 · PreprintNeuroscience