BioengineeringNature CommunicationsPeer-reviewed
A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD
Tristan X. McCallister, Colin K.W. Lim, Mayuri Singh…·Thomas 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.
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