Muscular dystrophy
- The use of genetically humanized animal models for personalized medicine approaches
Summary: Personalized medicine approaches benefit from humanized animal models. Here, we outline the usefulness, caveats and considerations for generating and using these models for pre-clinical studies of Duchenne muscular dystrophy.
- The roles of dystroglycan in the nervous system: insights from animal models of muscular dystrophy
Summary: Dystroglycan is a ubiquitous matrix receptor linked to brain and muscle disease. Unraveling the functions of this protein will inform basic and translational research on neural development and muscular dystrophies.
- Muscle strength deficiency and mitochondrial dysfunction in a muscular dystrophy model of Caenorhabditis elegans and its functional response to drugs
Editor's choice: Dystrophin-deficient Caenorhabditis elegans have measurably weak muscle strength and mitochondrial dysfunction, and they respond to drug treatments standard in treating human Duchenne muscular dystrophy.
- Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery
Summary: This work provides proof of principle for the use of myotonic dystrophy 1 patient-specific induced pluripotent stem cells to model muscle pathology in vitro and in drug discovery.
- A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9
Summary: The DMD KO rabbit engineered by CRISPR genome editing faithfully recapitulates the DMD pathologies, and could be a valuable tool for basic and translational studies to combat this disease.