Jerry Mendell, MD, director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital, and his fellow researchers, are the latest recipients of the Annals of Neurology prize for an outstanding contribution to clinical neuroscience. The study which helped Dr. Mendell and his team receive this award is titled, â€œSustained Î±-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
The study, published in the November 2010 issue of the Annals of Neurology, is part of multi-phase, first-ever successful gene therapy trial in muscular dystrophy, showing sustained expression of a transferred gene to muscle. The findings provide proof of principle that gene replacement to restore missing proteins in muscular dystrophy is a viable approach to therapy for this devastating disease. The follow up to this line of attack is gene replacement through the circulation and Dr. Mendell and his team of researchers have been awarded an NIH grant of $1,041,296 to continue their work.
This annual prize is given in recognition of a particularly novel and impactful contribution to our understanding of the mechanism or treatment of human diseases of the nervous system.