Open access
Aug 2026
Compact type II-C Cas9 nucleases with expanded PAM access and high fidelity for therapeutic genome editing
Three previously uncharacterized compact type II-C Cas9 orthologs are identified, and all show a strong deletion-biased repair signature and no detectable editing across 33 predicted off-target sites, expanding the CRISPR targeting space for AAV-deliverable therapeutic editing.
Qiaochu Wang, Sivakrishna Rao Gundra, Rashid Aman et al.
· bioRxiv · 0 citations