Dr. Rita Perlingeiro
University of Minnesota (Minneapolis, MN)
This grant will support a stem-cell therapy project using genetically-corrected LGMD2I patient cells. In this project, induced pluripotent stem cells (iPS cells) from LGMD2I patients will first be corrected for their genetic defect using novel recombinant DNA techniques. Corrected cells will be then induced to become muscle progenitor cells and will be transplanted into the muscles of immunocompromised FKRP-mutant mice. Transplanted mice will be analyzed for improvement in function and muscle strength.
This is the first time that a stem cell therapy is being tested in a model of LGMD2I.