The Next Generation of Mouse Model Preparation Platform
DMDhE51delE52hE53
| Strain Name | C57BL/6N-DMD<sup>hE51delE52hE53</sup>/MCL |
|---|
Nucleic Acid Drug R&D (Core Application)
Disease Mechanism and Pathology Research
Combination Therapy Evaluation
Gene Editing Therapy (CRISPR Technology Validation)
Biomarker Development
The model precisely replaces mouse Dmd exon 52 by knockout and replaces mouse exons 51 (mE51) and 53 (mE53) along with adjacent sequences with human exons 51 (hE51) and 53 (hE53) corresponding sequences, respectively, through precise gene editing. Sanger sequencing of RT-PCR products verified the successful transcript splicing of mE50-hE51-hE53-mE54. Meanwhile, this design simulates frame-shift mutations caused by common human DMD exon deletions, introducing a premature stop codon in the hE53 region, which results in the absence of functional dystrophin expression. This model preserves the specificity for drug screening targeting human DMD gene targets while reproducing the core pathological phenotypes of DMD, serving as an ideal tool for studying exon skipping therapies, gene editing repair, and evaluating the preclinical efficacy of related drugs. The Velogene DmdhE51delE52hE53 mouse model exhibits typical Duchenne muscular dystrophy (DMD) features, including dystrophin deficiency, muscle fiber degeneration, necrosis, decreased muscle strength, and elevated serum creatine kinase (CK) levels, successfully recapitulating the hallmark disease characteristics of DMD patients.
High Humanization of Drug Targets with High Translational Value: This model replaces key regions of the mouse Dmd gene with human DMD homologous sequences (hE51/hE53), precisely replicating human drug targets. Compared to traditional mdx mice, this model largely eliminates interference caused by interspecies gene sequence differences, significantly improving the clinical translation rate of preclinical pharmacodynamic evaluations targeting human-specific sites (such as ASO and sgRNA binding sites).
Precise Simulation of Clinical Hotspot Mutation Types: The model design simulates the most common human DMD exon deletion mutations (such as Δexon51) by introducing a frame-shift mutation and a premature termination codon (PTC), resulting in the complete absence of functional dystrophin. This enables it to faithfully reflect the core molecular pathological mechanism of DMD, serving as an ideal platform for validating “read-through” or “exon skipping” strategies to correct reading frame errors.
Dual Advantages of Molecular Validation and Phenotypic Stability: Verified by Sanger sequencing, this strain features a stable genetic background and achieves correct transcript splicing of mE50-hE51-hE53-mE54, ruling out false-positive results caused by aberrant splicing. Meanwhile, due to the lack of protein expression, the model stably reproduces typical DMD pathological phenotypes (such as elevated creatine kinase, muscle fiber necrosis and regeneration, and fibrosis), facilitating objective therapeutic efficacy evaluation.
Broad Applicability for Drug Screening: Benefiting from its humanization modification, this model is not only suitable for antisense oligonucleotide (ASO)-mediated exon skipping therapy research but also applicable for preclinical proof-of-concept (PoC) validation of various intervention approaches, including CRISPR/Cas9 gene editing (such as base editing and prime editing), RNA interference, and small molecule drugs, exhibiting extremely high versatility.
Pharmacodynamic Evaluation in an Immunocompetent Background: This model is typically constructed on an immunocompetent mouse background (such as C57BL/6), avoiding the impact of immunodeficiency on drug delivery efficiency and the muscle microenvironment. This enables researchers to examine the long-term therapeutic efficacy of drugs alongside potential immunogenicity risks within an intact immune system environment.
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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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.