U6-(gRNA)-(pro)-CBE-NT-bGHpA + EFS-CBE-CT-bGHpA
Two-vector system delivering CBE N-terminal and C-terminal split-intein fragments for reconstituted BE3 cytosine base editing
Characteristics
First AAV delivers CBE N-terminal (APOBEC1 + SpCas9 aa1-573) under EFS promoter. Second AAV delivers CBE C-terminal (SpCas9 aa574-1368) under selected promoter with U6-gRNA and BsmBI cloning site. Split at E573/C574 using DnaE intein. Reconstitutes full BE3 activity in target cells. NGG PAM, editing window positions 4-8.
Custom Inserts
SpCas9 gRNA target
(20-23 bp)
Literature Sources
- Komor et al. (2016). Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature.
Available Products
No products currently available with this design. Contact us for custom orders.
Additional Information
Applications: In vivo cytosine base editing when dual-AAV co-transduction is feasible. Liver, muscle, CNS applications. When SpCas9 NGG PAM compatibility and established editing window are required for therapeutic target.