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hPCSK9 Mouse Model
| Starin Name | C57BL/6N‑Pcsk9<sup>tm1Cin(hPCSK9)</sup> |
|---|---|
| Strain Background | C57BL/6N |

- Hypercholesterolemia & Lipid Metabolism Research: Investigating the role of human PCSK9 in LDL-C regulation, familial hypercholesterolemia, and dyslipidemia.
- PCSK9 Inhibitor Development & Efficacy Assessment: Preclinical evaluation of anti-PCSK9 monoclonal antibodies, small-molecule inhibitors, siRNAs/antisense oligonucleotides, and CRISPR-based gene therapies.
- Combination Immuno-oncology Studies: Testing the synergistic efficacy of “PCSK9 inhibitor + Immune Checkpoint Inhibitor (e.g., anti-PD-1)” in tumor models.
- Drug Combination Evaluation: Assessing the lipid-lowering synergy of PCSK9 antibodies with statins or other antihyperlipidemic agents.
- Cardiovascular Disease Modeling: Establishing diet-induced or genetically modified atherosclerotic models on a human PCSK9 background.
PCSK9 (proprotein convertase subtilisin/kexin type 9) is a key protein regulating cholesterol metabolism. Inhibiting PCSK9 is an effective approach to enhance the efficacy of immune checkpoint inhibitors, and the combination treatment strategy of ‘PCSK9 inhibitors + immune checkpoint inhibitors’ has demonstrated excellent therapeutic efficacy in tumor immunotherapy experiments. Targeting PCSK9 to develop new inhibitors or gene therapies holds bright prospects in both cardiovascular disease treatment and tumor immunotherapy. hPCSK9 humanized mice are important models for the research and development of PCSK9 inhibitors or gene therapies.
VeloGene’s independently developed C57BL/6N-hPCSK9 humanized mice utilize the genomic sequence corresponding to the human PCSK9 transcript NM_174936.4 (5’UTR-3’UTR) as the humanized sequence to precisely replace the genomic sequence corresponding to the mouse mPCSK9 transcript NM_153565.2 (5’UTR-3’UTR). This design ensures the specific expression of the human PCSK9 gene in mice, providing an essential experimental model for studying PCSK9 in cholesterol metabolism and related diseases.
Key Features
- Full-Length Cognate Replacement: Entire mouse Pcsk9locus (5′UTR through 3′UTR) is precisely replaced with the human PCSK9genomic sequence (RefSeq NM_174936.4), preserving native promoter, enhancers, and 3′ UTR.
- Physiological Expression Pattern: Human PCSK9 is expressed with authentic tissue specificity and developmental timing, mirroring endogenous Pcsk9regulation.
- High Translational Relevance: Supports evaluation of human-specific targeted therapies (antibodies, gene editing, ASOs) that would not cross-react with murine Pcsk9.
- C57BL/6N Background: Established on the widely used C57BL/6N inbred background, compatible with standard diet-induced obesity/atherosclerosis and tumor models.
- Accurately Simulate Human Gene Expression Patterns: By performing in-situ replacement of the full-length human PCSK9 genomic sequence at the PCSK9 locus in C57BL/6N mice, it ensures that the expression pattern and tissue specificity of the human PCSK9 gene in mice are consistent with the endogenous ones.
- Meet Specific Targeted Therapy Research Needs: If the target site of a gene-targeted therapeutic drug is located at the promoter of the human sequence, VeloGene’s exclusively developed humanized mice featuring the human PCSK9 promoter plus the full-length gene can be utilized.

FAQ
Game-changing benefits?
While competitors highlight germline efficiency gains, shorter timelines and enhanced 3Rs animal welfare benefits for their technologies, these are merely incremental improvements over traditional approaches. In sharp contrast, our proprietary technology delivers fully pure, homogeneous lineages—every single cell of the mice is derived exclusively from totipotent ES cells, with guaranteed 100% germline transmission efficiency. To experience these unparalleled benefits firsthand, enquire about your custom mouse model project with us or order embryos for in-house validation at your facility.
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•Free initial design proposal with zero obligations.
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Who owns the mouse lP? Do l need a licence from Mingceler?
All model generation projects of Mingceler operate under a fee-for-service framework.
IP Ownership: All intellectual property rights related to custom mouse models, including derived organs, tissues, cells, and biological materials, are the sole and exclusive property of the Client.
Third-Party Transfer Permission: The Client may independently decide to retain, utilize, or commercialize their custom models project materials (e.g., targeting vectors, ES cells, mouse lines) without the need for prior consent from Mingceler.
Licensing Exemption: The Client has full autonomy over all uses of the custom models or their derivatives, including but not limited to commercialization, distribution to third parties, and publication involving model data. No written license from Mingceler is required for such uses.
