Custom C-to-G Base Editor (AAV)
Custom C-to-G Base Editor (AAV)
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Direct C-to-G transversion editing in single-AAV format for in vivo gene therapy. Enables unique base conversions inaccessible to adenine or cytosine base editors for correcting transversion mutations in liver, muscle, CNS, and other AAV-accessible tissues. Compact architecture eliminates dual-vector requirements while delivering novel editing capability for therapeutic applications requiring guanine installation at cytosine positions.
Payload
- CGBE Base Editor: C-to-G transversion base editor expanding beyond transition mutations. Enables targeted C-to-G conversion addressing 11-40% of disease SNPs.
- 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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