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Humanized Target Gene Disease Models

Generate CDS-humanized mouse models by replacing endogenous coding sequences with human CDS regions, enabling more clinically relevant target validation and efficacy studies while accelerating preclinical research and therapeutic development.
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.
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FAQs
Overview
How Are Target Gene-Humanized Mouse Models Generated?
Target gene humanization uses precise genome-editing strategies to replace selected mouse coding sequences with their human counterparts while preserving appropriate genomic context. The replacement may involve a specific exon, multiple exons, or the complete coding region, depending on the target, protein structure, and research requirements. This flexible approach allows the resulting model to express a human or partially humanized protein while maintaining endogenous expression patterns and tissue distribution.
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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Scalable colony expansion with full genotyping support
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Base Strain
Catalog Number
Catalog NumberNameBase StrainResearch ApplicationAction
C001339Alb-cre+/MYC+C57BL/6JCyaLiver 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.
C001326BALB/c-hCD3BALB/cAnCyaResearch on the immune system; T cell activation and antigen recognition studies; Research on immunosuppressive therapy for autoimmune diseases; Development and evaluation of CD3-targeted drugs.
I001047BALB/c-hCD3 (line2)BALB/cAnCyaResearch on the immune system; T cell activation and antigen recognition studies; Research on immunosuppressive therapy for autoimmune diseases; Development of CD3-targeted drugs.
C001522B6-hLPA (CKI) /Alb-creC57BL/6NCyaResearch on atherosclerosis, hyperlipidemia, thrombotic cardiovascular diseases, etc; Preclinical evaluation of human LPA-targeted drugs.
C001521B6-hLPA (CKI)C57BL/6NCyaResearch on atherosclerosis, hyperlipidemia, thrombotic cardiovascular diseases, etc.; Preclinical evaluation of human LPA-targeted drugs.
C001523B6-hCALCAC57BL/6JCyaDiscovery and screening of migraine drugs and therapies; Evaluation of the efficacy and safety of migraine drugs and therapies; Research on vascular biology and blood pressure regulation; Research on cell proliferation and apoptosis; Research on tumor growth inhibition and inflammation; Research on the generation and differentiation of hematopoietic stem/progenitor cells.
I001220B6-hPCSK9/Apoe KOC57BL/6CyaDevelopment, screening, and preclinical evaluation of PCSK9-targeted drugs; Research on metabolic diseases such as hyperlipidemia, stroke, coronary heart disease, familial hypercholesterolemia (FH), and other atherosclerotic cardiovascular diseases (ASCVD).
C001713B6-hIL2RAC57BL/6NCyaIL2RA-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.
C001684B6-hFGFR1cC57BL/6NCyaScreening, 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).
C001419B6-hCD47C57BL/6JCyaDevelopment 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.
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FAQs
Frequently Asked Questions (FAQs)
What research areas are supported by target gene-humanized mouse models?
Target gene-humanized mouse models are widely used in oncology, immunology, metabolic disease, inflammation, cytokine biology, cardiovascular research, neuroscience, and rare disease studies. The appropriate model depends on the target's biological function, tissue expression, disease relevance, and interaction with the therapeutic candidate.
Why are humanized target models important for preclinical drug development?
Many antibodies, biologics, gene therapies, and other targeted treatments are designed to recognize human proteins and may not interact effectively with the corresponding mouse targets. Humanized target models help overcome this species-specific limitation, supporting more relevant target engagement, pharmacodynamic, efficacy, and safety-related studies.
Can only part of a target gene be humanized?
Yes. Depending on the research objective, humanization may involve a specific exon, functional domain, multiple coding exons, or the complete coding sequence. The humanization strategy should consider protein structure, therapeutic binding sites, endogenous gene regulation, and the biological function that must be preserved.
How do I select the right target gene-humanized mouse model?
Model selection should be based on the therapeutic modality, human-specific binding region, disease indication, required genetic background, and planned experimental endpoints. Researchers should also confirm the humanized sequence, protein expression, tissue distribution, and available validation data before beginning a preclinical study.
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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Global Antibody Drug Industry Development BlueBook (Frost & Sullivan)
Key Insights
The industry is undergoing a rapid transformation driven by next-generation modalities, globalized markets, and upstream technological innovations.
  • Market Structural Shift: Monoclonal antibodies drive steady growth, but ADCs and bispecifics are rapidly accelerating, reshaping the market with higher-value innovations.
  • Chinese Market Globalization: China is actively expanding globally, evidenced by a surge in high-value cross-border license-out deals.
  • Technology-Driven Efficiency: Advanced discovery engines—exemplified by Cyagen's HUGO-Ab platform and AI algorithms—are streamlining candidate screening, optimizing molecular design, and localizing the upstream supply chain.
  • Oncology-Focused Innovation: R&D pipelines remain heavily concentrated on high-incidence malignancies like non-small cell lung cancer, utilizing complex modalities to combat clinical resistance.
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