Dongsheng Duan, professor in the department of molecular microbiology & immunology at the University of Missouri in Columbia, has received an MDA grant totaling $527,670 over three years. The funds will help support Duan's continued research into gene therapy in Duchenne muscular dystrophy DMD).

DMD is caused by a mutation in the dystrophin gene, which leads to the absence, or near absence, of dystrophin protein.

Duan has worked extensively in the past with adeno-associated virus (AAV) "vectors," the emptied shells of viruses used to encapsulate healthy genes (or modified — in this case, shortened "mini-" or "mirco-dystrophin" genes), which are then injected locally or systemically into research model test subjects undergoing gene therapy treatment.

In previous studies, AAV-mediated micro-dystrophin gene therapy has shown great promise in ameliorating the symptoms of DMD, and recent developments in systemic AAV delivery suggest the potential for whole-body correction of the flawed dystrophin gene responsible for the disease.

In his latest work, Duan and colleagues plan to test both a new AAV delivery vehicle and a new micro-dystrophin gene, first in a research mouse model of DMD and later in another animal research model of the disease. The investigators will evaluate whether the novel combination ameliorates DMD symptoms.

Findings from this study potentially could accelerate DMD gene therapy research and help advance the strategy to human clinical testing.

"We can now cure a mouse with a DMD-like disease; however, this accomplishment has not been reproduced in people with the disease," Duan said. "The support we receive from MDA is essential for us to move DMD gene therapy forward."

Funding for this MDA grant began February 1, 2011.