Humanized Target Gene Disease Models

Q: Can models combine multiple genes (e.g., CD47 + SIRPα)?

Multiplex Humanization: Models like B6-hCD47/hSIRPα demonstrate 4.7-fold increased tumor clearance with dual blockade. Conditional Systems: Tissue-specific co-expression (e.g., Tet-On) ensures <2% leaky expression.

Q: How does TurboKnockout-Pro® minimize mosaicism in sickle cell models?

High-Fidelity Editing: eSpCas9(1.1) reduces off-target effects by >95%. ESC Cloning: Achieve >90% germline transmission and screen for variants via 30x WGS.

Q: How is the mouse strain selected for rare neurological disorders?

Mechanism-Driven Choice: Prioritize strains like C57BL/6J-SOD1 G93A for ALS. Genetic Purity: Backcross humanized alleles into >98% pure backgrounds. Phenotypic Screening: Pre-screen mouse strains to identify clinically relevant modifiers.

Q: How do you validate human protein expression levels (e.g., PD-L1)?

Transcriptomic: NanoString® analysis confirms mRNA within ±15% of human baselines. Proteomic: Flow cytometry benchmarks PD-L1 density to clinical samples (2,000–5,000 molecules/cell). Functional: Anti-PD-1 response correlation of R²=0.78 with clinical data.

Q: Can you share CAR-T toxicity data from models like B6-hCD47?

Off-Target Effects: B6-hCD47 models showed CRS Correlation: Humanized IL6/IL6R models achieved 89% alignment between murine cytokine spikes and clinical CRS grading.

Q: How do you ensure humanized gene functionality in complex diseases like leukemia?

Our multi-tiered validation strategy ensures functional fidelity: Pathway Alignment: Retain murine regulatory elements (e.g., promoters) while replacing coding sequences with human counterparts, mitigating interspecies discrepancies in pathways like JAK-STAT. Functional Testing: Validate via CAR-T efficacy assessments and PDX engraftment, as demonstrated in RIKEN’s ALL studies. Receptor-Ligand Compatibility: Co-humanize interacting pairs (e.g., PD-1/PD-L1) to preserve binding affinity.

Transform uncertainty into genetically precise solutions. Engineer humanized targets for CAR-T, PD-1, or hematologic studies with TurboKnockout® technology—accelerating your path to actionable insights. 

Clinically Validated Systems

Streamline testing of oncology, immunotherapies, and hematologic drugs in pre-validated, humanized environments.

Accelerated Model Development

Achieve 50% faster model delivery through gene-based engineering, outpacing traditional methods.

Custom Human Gene Integration

Seamlessly replace animal genes with human sequences to replicate disease mechanisms with unmatched accuracy.

Overview

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FAQs

Overview

Accelerate Therapeutic Validation with Human-Relevant Precision

Leverage cutting-edge gene-engineered models powered by TurboKnockout® to validate CAR-T, PD-1, and hematologic therapies in systems that mirror human biology—translating results into clinically actionable insights.

Explore Ready-to-Use Mouse Models

Discover over 18,000 validated mouse strains—including knockout, conditional knockout, and humanized models—covering 20+ research areas such as oncology, neurology, and metabolism. All models are supported by detailed genotype data and guaranteed quality, helping you fast-track discovery with confidence.

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Base Strain

Catalog Number

Catalog Number	Name	Base Strain	Research Application
C001339	Alb-cre+/MYC+	C57BL/6JCya	Liver Cancer Research: The model overexpresses the MYC oncogene in the liver, spontaneously developing liver cancer, thus serving as a tool to study the mechanisms of liver cancer initiation and progression. Drug Screening and Evaluation: Utilized to assess the efficacy of new anti-liver cancer drugs and therapeutic strategies, particularly those targeting MYC-driven tumors. Gene Function Studies: Investigates the role of the MYC gene in hepatocytes and its involvement in tumor formation. Transcriptional Regulation Studies: Explores the mechanisms of MYC in transcriptional regulation and its interactions with other genes and signaling pathways.
C001592	B6-hMASP2	C57BL/6NCya	MASP2-targeted drug screening, development, and evaluation; Research on the pathological mechanisms and therapeutic approaches of autoimmune and infectious diseases.
C001713	B6-hIL2RA	C57BL/6NCya	IL2RA-targeted drug screening, development, and evaluation; Research on the pathological mechanisms and therapeutic approaches of autoimmune diseases such as multiple sclerosis, type 1 diabetes, and rheumatoid arthritis. Tumor immunology and other anti-tumor research.
C001711	B6-hBAFFR (hTNFRSF13C)	C57BL/6NCya	TNFRSF13C-targeted drug screening, development, and evaluation; Research on the pathological mechanisms and therapeutic approaches of immune-mediated disorders such as common variable immunodeficiency (CVID), systemic lupus erythematosus (SLE), and Sjögren's syndrome; Research on the pathological mechanisms and therapeutic approaches of certain B cell malignancies.
C001684	B6-hFGFR1c	C57BL/6NCya	Screening, development, and preclinical efficacy evaluation of FGFR1c-targeted drugs; Study of pathological mechanisms and therapeutic methods for cancers and metabolic diseases such as obesity, diabetes, and metabolic-associated steatohepatitis (MASH).
C001419	B6-hCD47	C57BL/6JCya	Development and screening of targeting CD47 inhibitors/antibody drugs； Evaluation of the efficacy and safety of targeting CD47 inhibitors/antibodies； Evaluation of tumor immunotherapy and research on tumor immune escape mechanisms.
C001521	B6-hLPA (CKI)	C57BL/6NCya	Research on atherosclerosis, hyperlipidemia, thrombotic cardiovascular diseases, etc.; Preclinical evaluation of human LPA-targeted drugs.
I001187	B6-hDPP4 (line1)	C57BL/6NCya	Development of DPP4 inhibitor therapies; Research on Middle East Respiratory Syndrome (MERS-CoV) infection; Research on Severe Acute Respiratory Syndrome (SARS-CoV) infection.
I001188	B6-hDPP4 (line 2)	C57BL/6JCya	Development of DPP4 inhibitor therapies; Research on Middle East Respiratory Syndrome (MERS-CoV) infection; Research on Severe Acute Respiratory Syndrome (SARS-CoV) infection.
I001189	BALB/c-hDPP4 (line 2)	BALB/cAnCya	Development of DPP4 inhibitor therapies; Research on Middle East Respiratory Syndrome (MERS-CoV) infection; Research on Severe Acute Respiratory Syndrome (SARS-CoV) infection.

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FAQs

Frequently Asked Questions (FAQs)

Can models combine multiple genes (e.g., CD47 + SIRPα)?

How does TurboKnockout-Pro® minimize mosaicism in sickle cell models?

How is the mouse strain selected for rare neurological disorders?

How do you validate human protein expression levels (e.g., PD-L1)?

Can you share CAR-T toxicity data from models like B6-hCD47?

How do you ensure humanized gene functionality in complex diseases like leukemia?

Citation Database

Molecular Therapy: Methods & Clinical Development, March, 2025

Intracranial AAV administration dose-dependently recruits B cells to inhibit the AAV redosing

【Other】

Gut, February, 2025

E-twenty-six-specific sequence variant 5 (ETV5) facilitates hepatocellular carcinoma progression and metastasis through enhancing polymorphonuclear myeloid-derived suppressor cell (PMN-MDSC)-mediated immunosuppression

【Other】

Cell Death & Disease, February, 2025

Mcm5 mutation leads to silencing of Stat1-bcl2 which accelerating apoptosis of immature T lymphocytes with DNA damage

【Other】

Molecular Therapy, February, 2025

Single-cell data-driven design of armed oncolytic virus to boost cooperative innate-adaptive immunity against cancer

【Other】

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