Coalition to Cure Calpain 3 (C3) is excited to announce that we are funding a new research project with Dr. Rita Perlingeiro, Professor of Medicine and Lillehei Professor in Stem Cell and Regenerative Cardiovascular Medicine at the University of Minnesota. The project is titled “Gene editing of Calpain 3 in LGMD2A iPS cells.”
There has been tremendous excitement for the therapeutic potential of gene editing in treating genetic diseases. Dr. Perlingeiro specializes in the use of gene editing to correct induced pluripotent stem (iPS) cells from muscle diseases. These cells can then be transplanted into the muscles of models of muscular dystrophy.
The goal of the current project is to establish methods to utilize the gene editing technology CRISPR-CAS9 to genetically correct mutations in the Calpain 3 gene (CAPN3) in LGMD2A iPS cells. Gene correction will then be validated in vitro. These studies will determine the feasibility of a gene edited iPS-cell based stem cell therapy.
C3 Scientific Director Dr. Jennifer Levy notes, “Scientists are very excited about the possibility of ‘fixing’ disease-causing mutations with gene editing. In this project, Dr. Perlingeiro proposes to develop a strategy to correct mutations in the gene for Calpain 3 in patient-derived cells.”
Dr. Perlingeiro comments, “We are thrilled to receive the support from the C3 foundation on this important project. Gene editing technologies combined with the derivation of skeletal muscle progenitors from patient-specific pluripotent stem cells hold great therapeutic potential, and we hope to make an impact in the lives of LGMD2A patients”.