Custom Adenine Base Editor (AAV)
Custom Adenine Base Editor (AAV)
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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.
- SaABE8e: 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 pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- SaABE8e-V106W: SaABE8e variant with V106W mutation in TadA* domain, reducing off-target RNA editing while maintaining DNA editing efficiency.
- bGH pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- SaKHH-ABE8e: Expanded-PAM variant of SaABE8e with KKH mutations (E782K/N968K/R1015H) enabling NNNRRT PAM recognition.
- bGH pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- CjABE8e: 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 pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- Nme2ABE8e: Ultra-compact adenine base editor using Nme2Cas9 with N4CC PAM, enabling targeting of A-rich sequences.
- bGH pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- SauriABE8e: Compact adenine base editor using S. auricularis Cas9, providing alternative PAM specificity for expanded targeting.
- bGH pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- Target-AID: Alternative cytosine base editor using sea lamprey cytidine deaminase. Narrower editing window (positions 2-4) with reduced cellular toxicity.
- bGH pA: Compact and highly efficient polyadenylation signal from bovine growth hormone gene, widely used in AAV and size-constrained vectors.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
Deliverables
- Titer: 1e13 VG/mL or higher
- Full Capsid Ratio: 0.85 or higher
- Delivery Timeline: 18–21 days
Specifications
Single stranded AAV produced in 293T cells and purified through two (2) rounds of CsCl ultracentrifugation and stored in 0.001% F-68 in DPBS with additional 150 mM NaCl. Ships on dry ice overnight.
This construct can be packaged in hundreds of AAV serotypes to target your specific tissue. We offer all standard serotypes (AAV1-9) plus specialized variants for muscle (MyoAAV-2A, MyoAAV-1A, AAVMyo), brain (PHP.eB, AAV9P31, AAV2-retro), and clinical applications (AAV2, Spark100). Other popular options include AAV-rh.74, AAV6, AAV6.2FF, and AAV3. Browse our complete serotype library or contact us for recommendations.
References
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