hDMD Mouse Model
| Strain Name | hDMD Mouse Model |
|---|---|
| Strain Background | C57BL/6N |
Applications
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Exon-skipping validation: Test ASOs for reading-frame restoration in patient-matched deletions.
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Gene therapy evaluation: Assess micro-dystrophin AAV vectors and immune reactivity.
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CRISPR/base-editing studies: Model patient-specific DMD corrections and quantify on-target repair.
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Pathophysiology research: Study dystrophin–DGC interaction, membrane stability, and nNOS localization.
Strain Description
The hDMD Mouse Model carries a targeted knock-in of human DMD genomic DNA at the endogenous mouse locus. It replaces the murine Dmd sequence with human counterparts while preserving native promoters and regulatory elements. Therefore, it ensures physiological expression of human dystrophin protein or defined truncations in the same tissues where the endogenous gene is normally expressed, primarily skeletal and cardiac muscle. This human DMD knock-in model produces human dystrophin that functions within the dystrophin–glycoprotein complex in a physiologically relevant manner.
In this hDMD model, dystrophin links the actin cytoskeleton to the extracellular matrix via the dystrophin–glycoprotein complex (DGC). It acts as a mechanical shock absorber during muscle contraction. Furthermore, dystrophin stabilizes nNOS localization at the sarcolemma, regulating local blood flow.
This humanized DMD model allows therapeutics to engage authentic human target sequences. For example, you can test antisense oligonucleotides (AONs) designed to skip mutated exons. Similarly, it supports evaluation of CRISPR base-editing or micro-dystrophin AAV vectors against human epitopes. Consequently, the hDMD Mouse Model provides a translational platform for DMD drug efficacy and safety assessment that conventional models cannot offer.
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.
How much for a project assessment?
•Free initial design proposal with zero obligations.
•Request a free quote!
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.