Alireza Edraki

This study removes a hexanucleotide repeat expansion in C9ORF72 using CRISPR/Cas9 in cell and animal models, reducing disease hallmarks.

This study shows that 5' modifications to DNA repair templates enhance precision genome editing efficiency in multiple animal models and human cells.

The paper demonstrates that NmeCas9, a Cas9 ortholog, exhibits high-fidelity genome editing in human cells with minimal off-target effects and multiple PAM compatibility.

The paper identifies and characterizes two new anti-CRISPR protein families that inhibit type II-C Cas9 systems, showing potent inhibition of NmeCas9 and cross-species activity.