Posted February 26, 2013
Paul Martin, Ph.D., Research Institute at Nationwide Children's Hospital
A promising therapeutic avenue for DMD is the use of gene therapy to overexpress GALGT2, an enzyme that alters skeletal muscle glycosylation to boost the expression of proteins that ameliorate disease. Recent studies suggest that overexpression of Galgt2 in skeletal muscle can inhibit DMD disease progression in several muscular dystrophy animal models. Therefore, Dr. Paul Martin at the Research Institute at Nationwide Children's Hospital has developed gene therapy vectors for human use to deliver the GALGT2 gene to skeletal and/or cardiac muscle. Dr. Martin's research team will complete dose response studies in two DMD animal models that are necessary to move this novel therapeutic intervention into a clinical trial. The long-term goal Dr. Martin's research is to develop a systemic treatment for all the muscles of a DMD patient.