U6-(gRNA)-(pro)-SaKKH-ABE8e-bGHpA

Express sgRNA under U6 and SaKKH-ABE8e under selected promoter for expanded NNNRRT PAM targeting scope

Characteristics

KKH mutations (E782K/N968K/R1015H) expand PAM recognition from NNGRRT to NNNRRT, significantly increasing targetable sites. Achieved highest therapeutic editing in Davis 2022 study with 93% knockdown of PCSK9/Pcsk9/Angptl3. Superior on-target activity compared to standard NNGRRT PAM variant. Editing window at positions 4-9.

Custom Inserts

SaCas9-KKH gRNA Target (21-23 bp)

Available Products

Additional Information

Applications: Maximum targeting flexibility with single-AAV SaCas9 platform. Preferred variant for therapeutic applications requiring robust editing. Validated for lipid-lowering gene therapy via PCSK9 and ANGPTL3 disruption. Use when target sites contain NNNRRT PAMs that exclude standard NNGRRT sequences.