Custom Adenine Base Editor (Dual AAV)
Custom Adenine Base Editor (Dual AAV)
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Precision A-to-G base editing via dual-AAV split-intein delivery for in vivo applications. SpCas9-based ABE7.10 system reconstitutes in target cells from co-transduced N-terminal and C-terminal AAV vectors. Ideal for liver, muscle, and CNS applications requiring adenine editing with established SpCas9 PAM compatibility (NGG) and broad targeting scope. Alternative to single-AAV compact editors when NGG PAM access is essential.
Payload
- ABE N-term: N-terminal fragment of ABE7.10 split via Npu intein at E573. Requires C-terminal counterpart for reconstitution.
- ABE C-term: C-terminal fragment of ABE7.10 split via Npu intein at C574. Pairs with N-terminal fragment for editor reconstitution.
- EFS Promoter: Compact ubiquitous constitutive promoter derived from a truncation of full-length EF1α, retaining stable long-term expression with significantly lower epigenetic silencing than CMV in dividing cells and in vivo. Widely used in size-constrained vectors including AAV and lentiviral systems.
- bGH PolyA Signal: Compact (225 bp) polyadenylation signal from bovine growth hormone with 3-fold higher expression than SV40 early polyA in key mammalian cell types. Size advantage over SV40 polyA makes it the preferred terminator for AAV vectors with limited cargo space. Standard terminator in commercial gene therapy and lentiviral vector platforms with decades of in vivo validation.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
Specifications
Single-stranded AAV produced in HEK293T cells via triple transfection and purified by two rounds of CsCl ultracentrifugation. Titer determined by qPCR. Full capsid content ≥85% by mass photometry. Endotoxin ≤1 EU per 1×10¹³ VG. Purity ≥95% by SDS-PAGE. Formulated in DPBS supplemented with 150 mM NaCl and 0.001% Pluronic F-68. Store at −80°C long-term. Stable at −20°C for up to 2 months and at 4°C for 1–2 weeks after thaw.
Deliverables
- Purified AAV at 1e13 VG/mL or higher in selected volume
- Full capsid ratio 0.85 or higher
- Batch report (titer, full/empty ratio, endotoxin, SDS-PAGE)
- Payload sequence file
- Ships on dry ice
References
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