NeurosciencebioRxivPreprint
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…·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.
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