U6-(gRNA)-(pro)-SaABE8e-bGHpA

Express sgRNA under U6 and SaABE8e under selected promoter for single-AAV adenine base editing with NNGRRT PAM

Characteristics

Achieves 66% liver, 33% heart, and 22% muscle editing in vivo. Demonstrates 2.5-fold higher efficiency than dual-AAV split-intein systems at matched doses. NNGRRT PAM recognition. Editing window at protospacer positions 4-9. Compact SaCas9 architecture (~4.5 kb total) fits in single AAV with room for tissue-specific promoters.

Parts

SaABE8e bGH pA U6 Promoter

Custom Inserts

SaCas9 gRNA Target (21-23 bp)

Literature Sources

  1. Davis et al. (2022). Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors. Nat Biomed Eng.

Available Products

Additional Information

Applications: Hard-to-transduce tissues including liver, heart, and muscle where dual-AAV co-transduction is limiting. Validated for therapeutic PCSK9 editing with 93% protein knockdown and substantial plasma cholesterol reduction. Use when single-AAV delivery is required and target site contains NNGRRT PAM downstream of target adenine.