The LGMD2I Research Fund has recently awarded a grant to 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. The project will be led by Dr. Rita Perlingeiro, at the University of Minnesota, who developed groundbreaking techniques to obtain skeletal muscle progenitors out of patients’ stem cells.
This will be the first time that a stem cell therapy is tested in a mouse model of LGMD2I. If encouraging results are obtained, further testing will be done in order to bring this potential therapeutic approach to the clinic.