A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD
T. X. McCallister, C. K. W. Lim, W. M. Terpstra, M. A. Z. C., S. Zhang, J. E. Powell, T. Gaj
This study develops a high-fidelity CRISPR-Cas13 system that reduces C9ORF72 repeat RNA expression in a rodent model, decreasing RNA foci and reversing transcriptional deficits without affecting normal C9ORF72 levels.
bioRxiv · Dec 12, 2023Bioengineering