Hansell Stedman, associate professor of surgery at the University of Pennsylvania in Philadelphia, was awarded an MDA research grant totaling $300,000 over a period of three years to study early immune responses during administration of gene therapy.
Gene therapy is a promising treatment strategy for Duchenne muscular dystrophy (DMD) and other muscle diseases, but it has been hindered by the body’s immune response. Genes can be delivered via virus-like particles (viral vectors), but the immune system can identify them as foreign, and mount a response that can inactivate the vector and cause inflammation in the muscle. Stedman plans to study the very earliest phases of the immune response, combining approaches gleaned from immunology, surgical critical care, genetics and virology.
“This mechanistic information will identify rational targets for transient immunosuppression prior to vector administration,” Stedman says, “thereby improving the chances for safe and durable therapy for these devastating childhood-onset diseases.” His work will be performed in the mouse model of DMD.
“In our view, the entire field is tantalizingly close to the development of gene therapy for Duchenne and other forms of muscular dystrophy, but we must explore a number of strategies to address the remaining obstacles,” he says. “Understanding how the innate immune system recognizes injured cells should improve our chances for a successful intervention.”
Funding for this MDA grant began August 1, 2013.