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Animal Models for Duchenne Muscular Dystrophy

One key aspect of Duchenne muscular dystrophy (DMD) research is the use of animal models, which have played a critical role in advancing our understanding of DMD pathogenesis and evaluating potential therapeutic strategies. Our company is committed to providing a range of disease-modeling services to support researchers in investigating DMD and developing potential treatment methods. Our advances in molecular biology allow us to help our clients generate a variety of animal models for DMD research, including murine, canine, and feline DMD animal models.

Background

DMD is a devastating X-linked genetic rare disease with a high prevalence of 1 in 3500 live births. It is caused by a mutation in the gene DMD encoding dystrophin, resulting in the loss of the cytoskeletal protein dystrophin. The disease is characterized by progressive muscle weakness and wasting, leading to walking disability and premature death. Research on mutations in the DMD gene has laid the foundation for research on DMD pathogenesis and therapeutic strategies, and the latter must rely on DMD animal models. Currently, there are more than 60 experimental animal models of DMD, the most commonly used being murine and canine models. Based on these models, a variety of therapies have been developed, such as gene therapy, cell therapy, exon skipping, and read-through therapy.

Fig. 1 A male canine with X-linked muscular dystrophy (CXMDJ) at 6 months of age.

Fig. 1 A male canine with X-linked muscular dystrophy (CXMDJ) at 6 months of age. (Nakamura A, et al., 2011)

Disease Modeling Services

One advantage of our disease modeling services is the ability to customize animal models to specific research needs. This allows researchers to study the effects of different mutations and levels of dystrophin on DMD pathogenesis and potential treatments. We have generated a variety of animal models for DMD research, such as worm, fish, dog, cat, rat, pig, mouse, and monkey models. We are proficient in precise genome editing techniques, such as CRISPR/Cas9 and TALEN. And genetic interventions range from point mutations to complete deletion of the largest gene—DMD gene, double knockouts, and humanized constructs. We offer animal models including but not limited to:

  • Murine DMD models
    • Mdx mouse and its genetic variants mdx2cv, mdx3cv, mdx4cv, mdx5cv, mdx52 and DMDnull
    • Double knockout mice, including mdx-utrn−/− mouse, mdx/Cmah−/− mouse, and mdx4cv/mTRko mouse
    • Dmd-mutated Wistar-Imamichi rats
  • Canine DMD models
    We provide several different DMD canine models with mutations in the DMD gene for studying the effects of potential therapies on large muscles, such as those in the limbs and back.
    • Golden Retriever muscular dystrophy (GRMD)
    • Canine X-linked muscular dystrophy (CXMDJ)
    • Cavalier King Charles Spaniels with muscular dystrophy (CKCS-MD)
  • Feline DMD models
    Hypertrophy feline muscular dystrophy (HFMD)
  • Other DMD animal models
    • DMD-modified Diannan miniature pigs
    • DMD KO rabbits
    • Rhesus monkeys
  • Non-mammalian DMD models
    • Zebrafish dmdta222a mutants with dystrophin deficiency
    • Dystrophin-deficient Drosophila melanogaster
    • Caenorhabditis elegans DMD model with loss-of-function in dystrophin homolog gene dys-1

Our company's disease modeling services and expertise in animal models for DMD provide a significant advantage to researchers studying this disorder. We are committed to supporting and advancing DMD research through our services and expertise. If you are interested in our animal modeling services, please contact us for more information.

Reference

  • Nakamura, A.; Takeda, S. Mammalian models of Duchenne Muscular Dystrophy: pathological characteristics and therapeutic applications. Journal of Biomedicine and Biotechnology, 2011.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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