University of Florida Health Researchers Gain New Insight Into Muscular Dystrophy Gene

University of Florida Health researchers are gaining new insights into cells linked to a genetic mutation that causes a type of muscular dystrophy.

Using genome editing, the researchers developed mice that replicate the developmental timing of a dysfunctional gene that causes myotonic dystrophy type 1, or DM1. They then showed that two types of cells — precursor cells needed for muscle regeneration and epithelial cells in the brain that help produce cerebral spinal fluid — are particularly sensitive to the DM1 mutation.

The findings were published in the journal Genes & Development by a research team led by Maurice Swanson, Ph.D., a professor in the UF College of Medicine’s Department of Molecular Genetics and Microbiology and associate director of the UF Center for NeuroGenetics.

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Learn more about the technology available for licensing: Dmpk CTG480 Knock-in Mouse Model for Myotonic Dystrophy Type 1.