CRISPR-Cas3 for ATTR: When “Deleting” Beats “Cutting”
Researchers from the University of Tokyo led by Tomoji Mashimo and Kazuto Yoshimi (IMS-UTokyo) have shown that CRISPR-Cas3 editing can inactivate the TTR gene via long, predominantly directional deletions, with robust efficacy in mouse liver and a highly favorable safety profile (off-target/IFM) compared to Cas9.