1
/
of
1
Custom Adenine Base Editor (AAV)
Custom Adenine Base Editor (AAV)
SKU:
Regular price
$1,995
Regular price
Sale price
$1,995
Unit price
/
per
Selected promoter is too long for this design
Couldn't load pickup availability
Precision A-to-G base editing in single-AAV format for in vivo gene therapy and hard-to-transduce cells. Choose from compact ABE8e variants (SaCas9, SaCas9-KKH, CjCas9, Nme2Cas9, SauriCas9) with complementary PAM specificities collectively targeting ~82% of genomic adenines. Ideal for liver, heart, muscle, and CNS applications requiring high editing efficiency without dual-AAV co-transduction constraints. Validated for therapeutic development and systemic delivery.
Payload
U6-(gRNA)-(pro)-SaABE8e-bGHpA: Express sgRNA under U6 and SaABE8e under selected promoter for single-AAV adenine base editing with NNGRRT PAM. Uses the following parts:
- SaABE8e Base Editor: Compact adenine base editor using S. aureus Cas9 (NNGRRT PAM). TadA8e deaminase provides 590-fold faster deamination than ABE7.10 with editing window at positions 4-9.
- 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.
U6-(gRNA)-(pro)-SaABE8e-V106W-bGHpA: Express sgRNA under U6 and SaABE8e V106W under selected promoter for minimized off-target RNA editing. Uses the following parts:
- SaABE8e-V106W Base Editor: SaABE8e variant with V106W mutation in TadA* domain, reducing off-target RNA editing while maintaining DNA editing efficiency.
- 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.
U6-(gRNA)-(pro)-SaKKH-ABE8e-bGHpA: Express sgRNA under U6 and SaKKH-ABE8e under selected promoter for expanded NNNRRT PAM targeting scope. Uses the following parts:
- SaKKH-ABE8e Base Editor: Expanded-PAM variant of SaABE8e with KKH mutations (E782K/N968K/R1015H) enabling NNNRRT PAM recognition.
- 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.
U6-(gRNA)-(pro)-CjABE8e-bGHpA: Expresses sgRNA under U6 and CjABE8e under selected promoter for ultra-compact single-AAV delivery. Uses the following parts:
- CjABE8e Base Editor: Ultra-compact adenine base editor using Campylobacter jejuni Cas9. Smallest ABE architecture (~3.8 kb) with 22-nt protospacer and editing window at positions 3-11.
- 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.
U6-(gRNA)-(pro)-Nme2ABE8e-bGHpA: Express sgRNA under U6 and Nme2ABE8e under selected promoter for A/T-rich region targeting with N4CC PAM. Uses the following parts:
- Nme2ABE8e Base Editor: Ultra-compact adenine base editor using Nme2Cas9 with N4CC PAM, enabling targeting of A-rich sequences.
- 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.
U6-(gRNA)-(pro)-SauriABE8e-bGHpA: Express sgRNA under U6 and SauriABE8e under selected promoter for alternative PAM specificity and genome coverage. Uses the following parts:
- SauriABE8e Base Editor: Compact adenine base editor using S. auricularis Cas9, providing alternative PAM specificity for expanded targeting.
- 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.
U6-(gRNA)-(pro)-Target-AID-bGHpA: Express gRNA under U6 and Target-AID under selected pro for narrow-window cytosine editing with reduced bystander mutations. Uses the following parts:
- Target-AID Base Editor: Alternative cytosine base editor using sea lamprey cytidine deaminase. Narrower editing window (positions 2-4) with reduced cellular toxicity.
- 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
Share
