Custom OpenCRISPR-1 Editor (Dual-AAV)
Custom OpenCRISPR-1 Editor (Dual-AAV)
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First AI-designed dual-vector CRISPR system. OpenCRISPR-1 (1380aa) matches SpCas9 on-target activity while reducing off-target editing by 95%. Compatible with canonical SpCas9 sgRNAs and existing dual-AAV workflows. Lacks immunodominant SpCas9 T cell epitopes, potentially reducing immunogenicity risk. NGG PAM with strict specificity - minimal activity at non-NGG sites enhances precision. Ideal for applications prioritizing specificity over promiscuous PAM recognition1,2.
- OpenCRISPR-1 Nuclease: First fully AI-designed CRISPR nuclease created by large language models trained on >1 million CRISPR operons. Comparable on-target activity to SpCas9 (56.4% vs 47.1% median indel rates) with 95% reduction in off-target editing (0.32% vs 6.1%). 403 mutations from SpCas9 and 182 mutations from any natural CRISPR protein. Compatible with canonical SpCas9 sgRNAs and standard base editing architectures. Lacks immunodominant SpCas9 T cell epitopes, suggesting reduced immunogenicity. Blunt-end double-strand breaks. Strict NGG PAM requirement provides enhanced specificity - minimal activity at non-NGG PAMs.
- EGFP: Gold-standard green fluorescent reporter (488/507 nm) engineered from Aequorea victoria GFP, with ~35-fold greater brightness than wild-type via F64L/S65T mutations. Excitation peak perfectly matched to 488 nm laser lines on standard confocal and flow cytometry systems. The default choice for gene expression reporters, protein localization, and live-cell imaging across virtually all model organisms.
- KASH Domain: The KASH domain is the conserved C-terminal transmembrane domain from nesprin/SYNE family proteins that anchors to the outer nuclear membrane (ONM). Defined by Starr & Han (2002). Used to tether proteins to the outer nuclear membrane in engineered constructs.
- CAG Promoter: Synthetic hybrid promoter composed of the CMV immediate-early enhancer and chicken β-actin promoter, enabling strong, ubiquitous expression across most mammalian cell types. Produces very high initial expression but may undergo silencing in certain tissues over long-term in vivo studies.
- 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.
- SV40 Late PolyA Signal: Most widely used polyadenylation signal in mammalian expression vectors, derived from SV40 late gene region.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- OpenCRISPR-1 Nuclease: First fully AI-designed CRISPR nuclease created by large language models trained on >1 million CRISPR operons. Comparable on-target activity to SpCas9 (56.4% vs 47.1% median indel rates) with 95% reduction in off-target editing (0.32% vs 6.1%). 403 mutations from SpCas9 and 182 mutations from any natural CRISPR protein. Compatible with canonical SpCas9 sgRNAs and standard base editing architectures. Lacks immunodominant SpCas9 T cell epitopes, suggesting reduced immunogenicity. Blunt-end double-strand breaks. Strict NGG PAM requirement provides enhanced specificity - minimal activity at non-NGG PAMs.
- mCherry: Obligate monomeric red fluorescent protein (587/610 nm) with best-in-class photostability among common red reporters, enabling long-term timelapse imaging without bleaching artifacts. Fast maturation (<1 hr at 37°C) with full tolerance to N- and C-terminal fusions. Most widely used red channel partner for EGFP in two-color mammalian cell imaging.
- CAG Promoter: Synthetic hybrid promoter composed of the CMV immediate-early enhancer and chicken β-actin promoter, enabling strong, ubiquitous expression across most mammalian cell types. Produces very high initial expression but may undergo silencing in certain tissues over long-term in vivo studies.
- 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.
- SV40 Late PolyA Signal: Most widely used polyadenylation signal in mammalian expression vectors, derived from SV40 late gene region.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- OpenCRISPR-1 Nuclease: First fully AI-designed CRISPR nuclease created by large language models trained on >1 million CRISPR operons. Comparable on-target activity to SpCas9 (56.4% vs 47.1% median indel rates) with 95% reduction in off-target editing (0.32% vs 6.1%). 403 mutations from SpCas9 and 182 mutations from any natural CRISPR protein. Compatible with canonical SpCas9 sgRNAs and standard base editing architectures. Lacks immunodominant SpCas9 T cell epitopes, suggesting reduced immunogenicity. Blunt-end double-strand breaks. Strict NGG PAM requirement provides enhanced specificity - minimal activity at non-NGG PAMs.
- EGFP: Gold-standard green fluorescent reporter (488/507 nm) engineered from Aequorea victoria GFP, with ~35-fold greater brightness than wild-type via F64L/S65T mutations. Excitation peak perfectly matched to 488 nm laser lines on standard confocal and flow cytometry systems. The default choice for gene expression reporters, protein localization, and live-cell imaging across virtually all model organisms.
- KASH Domain: The KASH domain is the conserved C-terminal transmembrane domain from nesprin/SYNE family proteins that anchors to the outer nuclear membrane (ONM). Defined by Starr & Han (2002). Used to tether proteins to the outer nuclear membrane in engineered constructs.
- CAG Promoter: Synthetic hybrid promoter composed of the CMV immediate-early enhancer and chicken β-actin promoter, enabling strong, ubiquitous expression across most mammalian cell types. Produces very high initial expression but may undergo silencing in certain tissues over long-term in vivo studies.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- OpenCRISPR-1 Nuclease: First fully AI-designed CRISPR nuclease created by large language models trained on >1 million CRISPR operons. Comparable on-target activity to SpCas9 (56.4% vs 47.1% median indel rates) with 95% reduction in off-target editing (0.32% vs 6.1%). 403 mutations from SpCas9 and 182 mutations from any natural CRISPR protein. Compatible with canonical SpCas9 sgRNAs and standard base editing architectures. Lacks immunodominant SpCas9 T cell epitopes, suggesting reduced immunogenicity. Blunt-end double-strand breaks. Strict NGG PAM requirement provides enhanced specificity - minimal activity at non-NGG PAMs.
- mCherry: Obligate monomeric red fluorescent protein (587/610 nm) with best-in-class photostability among common red reporters, enabling long-term timelapse imaging without bleaching artifacts. Fast maturation (<1 hr at 37°C) with full tolerance to N- and C-terminal fusions. Most widely used red channel partner for EGFP in two-color mammalian cell imaging.
- CAG Promoter: Synthetic hybrid promoter composed of the CMV immediate-early enhancer and chicken β-actin promoter, enabling strong, ubiquitous expression across most mammalian cell types. Produces very high initial expression but may undergo silencing in certain tissues over long-term in vivo studies.
- 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.
- SV40 Late PolyA Signal: Most widely used polyadenylation signal in mammalian expression vectors, derived from SV40 late gene region.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- OpenCRISPR-1 Nuclease: First fully AI-designed CRISPR nuclease created by large language models trained on >1 million CRISPR operons. Comparable on-target activity to SpCas9 (56.4% vs 47.1% median indel rates) with 95% reduction in off-target editing (0.32% vs 6.1%). 403 mutations from SpCas9 and 182 mutations from any natural CRISPR protein. Compatible with canonical SpCas9 sgRNAs and standard base editing architectures. Lacks immunodominant SpCas9 T cell epitopes, suggesting reduced immunogenicity. Blunt-end double-strand breaks. Strict NGG PAM requirement provides enhanced specificity - minimal activity at non-NGG PAMs.
- SV40 Late PolyA Signal: Most widely used polyadenylation signal in mammalian expression vectors, derived from SV40 late gene region.
- 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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