Duchenne Muscular Dystrophy (DMD) is one of the most frequent genetic diseases and affects one in 3,500 boys worldwide. DMD patients suffer from progressive muscle weakness and loss beginning in early childhood due to absence of the functional protein dystrophin required to maintain intact muscles. Life-threatening conditions such as heart disease or difficulty breathing can arise. Existing treatment options include using medications to promote muscle growth or to relieve chronic inflammation associated with DMD. However, these medications can have significant side effects and are not curative. Ongoing research on DMD treatment has shown that gene therapy is a promising strategy for recovering dystrophin function; however, a dangerous immune response in DMD patients associated with chronic inflammation that exists in dystrophic muscles as secondary pathology can occur after the treatment with gene vehicle. To address this problem, investigators at Pitt have developed a dual gene therapy that both restores dystrophin function and mitigates inflammatory response associated with chronic inflammation.
Yang, Q., Tang, Y., Imbrogno, K., Lu, A., Proto, J. D., Chen, A., Guo, F., Fu, F. H., Huard, J., & Wang, B. (2012). AAV-based shRNA silencing of NF-κB ameliorates muscle pathologies in mdx mice. Gene Therapy, 19(12), 1196–1204. https://doi.org/10.1038/gt.2011.207
Bing Wang, Juan Li, and Xiao Xiao. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. PNAS. Dec 5, 2000 97 (25) 13714-13719; https://doi.org/10.1073/pnas.240335297