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Custom Cytosine Base Editor (Dual AAV)
Custom Cytosine Base Editor (Dual AAV)
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$2,295
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Precision C-to-T base editing via dual-AAV split-intein delivery for in vivo applications. Choose from SpCas9-based (BE3) or SaCas9-based (saCBE, saBE3.9max) systems with standard or KKH-enhanced PAM recognition. Reconstitutes in target cells from co-transduced N-terminal and C-terminal AAV vectors. Ideal for liver, muscle, and CNS applications requiring cytosine editing with flexible PAM targeting (NGG, NNGRRT, or NNNRRT).
Payload
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. Uses the following parts:
- CBE N-term: N-terminal fragment of cytosine base editor split via Npu intein at E573. Requires C-terminal counterpart for reconstitution.
- CBE C-term: C-terminal fragment of cytosine base editor 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.
U6-(gRNA)-(pro)-SaCBE-NT-bGHpA + EFS-SaCBE-CT-bGHpA: Two-vector system delivering S. aureus CBE split-intein fragments with NNGRRT PAM recognition. Uses the following parts:
- SaCBE N-term: N-terminal fragment of SaCas9-based cytosine editor split via Npu intein at Lys534. Compact size enables efficient AAV packaging.
- SaCBE C-term: Compact SaCas9-based cytosine editor split via Npu intein for dual-AAV delivery. Smaller size than SpCas9 enables efficient packaging.
- 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.
U6-(gRNA)-(pro)-SaCBE-NT-bGHpA + EFS-SaCBE-KKH-CT-bGHpA: Two-vector system with SaCBE N-terminal and KKH-enhanced C-terminal for expanded NNNRRT PAM targeting. Uses the following parts:
- SaCBE-KKH C-term: SaCas9-KKH variant base editor with altered PAM recognition (NNNRRT). Enables broader targeting when combined with compact AAV packaging.
- SaCBE N-term: N-terminal fragment of SaCas9-based cytosine editor split via Npu intein at Lys534. Compact size enables efficient AAV packaging.
- 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.
U6-(gRNA)-(pro)-SaBE39max-NT-bGHpA + EFS-SaBE39max-CT-bGHpA: Two-vector system delivering optimized S. aureus BE3.9max split-intein fragments with codon-optimized APOBEC1. Uses the following parts:
- SaBE3.9max N-term: N-terminal fragment of optimized SaCas9 base editor split via Npu intein at Lys534. Combines compact size with enhanced expression.
- SaBE3.9max C-term: C-terminal fragment of optimized SaCas9 base editor split via Npu intein at Cys535. Pairs with N-terminal for high-efficiency editing.
- 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.
U6-(gRNA)-(pro)-SaBE39max-NT-bGHpA + EFS-SaBE39max-KKH-CT-bGHpA: Two-vector system with optimized SaBE3.9max N-terminal and KKH-enhanced C-terminal for maximum efficiency and PAM flexibility. Uses the following parts:
- SaBE3.9max N-term: N-terminal fragment of optimized SaCas9 base editor split via Npu intein at Lys534. Combines compact size with enhanced expression.
- SaBE3.9max-KKH C-term: Optimized SaCas9-KKH base editor with enhanced nuclear localization and NNNRRT PAM. Combines BE3.9max improvements with broader targeting.
- 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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