Progressive Muscular Dystrophy Models

Wild Mouse 750
Wild Mouse 750
Human Xenograft Models
Cell Derived Xenograft Models
Autoimmune Disease Models
Systemic Lupus Erythematosus ModelsMultiple Sclerosis ModelsRheumatoid Arthritis ModelsPsoriasis ModelsInflammatory Bowel Disease ModelsAtopic Dermatitis Models Asthma Models
Metabolic Disease Mouse Models
Obesity ModelsDiabetes ModelsNon-alcoholic Steatohepatitis (NASH) ModelsHyperlipidemia ModelsOsteoporosis ModelsHyperuricemia Models
Humanized Immune System Models
huPBMC-NCGhuHSC-NCG-XhuHSC-NCG-hIL15huHSC-NCG
Organs or Tissue Humanized Mouse Models
Wild Mouse 765
Wild Mouse 765
Rare Disease Mouse Models
Progressive Muscular Dystrophy ModelsHemophilia A Models
Neurodegenerative Disease Mouse Models
Alzheimer’s Disease (AD) ModelsParkinson’s Disease (PD) ModelsHuntington's Disease (HD) ModelsAmyotrophic Lateral Sclerosis (ALS) ModelsTransthyretin Amyloidosis (ATTR) Models
Immuno-Oncology Mouse Models
Human Xenograft ModelsHumanized Immune Checkpoint ModelsHumanized Immune System ModelsSpontaneous Tumor Models

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder involving the heart and respiratory muscles. The disease is characterized by progressive atrophy and weakness of the proximal skeletal muscles of the extremities, pseudohypertrophy of the gastrocnemius muscles of the lower legs. It can result in death at an average age of 27. The DMD gene encodes a long rod-shaped cytoskeletal protein, mainly localized on the inner surface of myofibers in skeletal and cardiac muscles. This anti-myotonic protein aids in maintaining the integrity and flexibility of muscle fibers during contraction and is a component of the myotonic dystrophy protein complex, which plays a crucial role in maintaining cellular structure. In humans, mutations in this gene can result in Duchenne-type and Becker-type myotonic dystrophy.

GemPharmatech has targeted the mouse DMD gene with gene editing technique (Dmd-KO, T049591) and generated frameshift mutation(s) of the DMD gene. These models can be used for screening muscular dystrophy therapeutics or pathophysiological studies of DMD.


Application

  • Screening of muscular dystrophy therapeutics

  • Pathophysiological studies of muscular dystrophy