MDA awarded a research grant totaling $390,000 over three years to Rita Perlingeiro, associate professor in the Department of Medicine at the University of Minnesota in Minneapolis. The funds will help support Perlingeiro's study of genetic correction and muscle regeneration in Duchenne (DMD) and Becker (BMD) muscular dystrophies.
"The practical and ethical problems associated with the use of embryonic stem cells have been eliminated with the revolutionary discovery of reprogramming somatic cells, such as skin, to a pluripotent state (iPS cells)," Perlingeiro explains. "This enables the generation of patient-specific induced pluripotent stem cells that could be used for transplantation." (Pluripotent cells can mature, or differentiate, into any cell type and can be expanded indefinitely in the lab.)
Perlingeiro and colleagues have invented and continue to refine a method to efficiently generate immature skeletal muscle cells from iPS cells. In addition, they have shown that, once transplanted, the cells integrate with existing skeletal muscle cells in an iPS cell model of DMD, and in cells taken from healthy mice and healthy humans.
Now, Perlingeiro is working to determine the efficiency with which genetically corrected iPS cells can increase muscle regeneration in DMD mice.
Perlingeiro's work could lay the groundwork for clinical development of pluripotent stem cells for muscular dystrophy.
"Because disease progression for DMD takes over a decade, there is sufficient time to generate and test iPS cells," Perlingeiro says, "making DMD an ideal candidate for early-stage iPS cell therapy clinical trials."
Funding for this MDA grant began Aug. 1, 2012.