PPMD has awarded a $50,000 Exploratory Grant to University of Missouri to test SERCA2A gene therapy as a potential treatment for dogs with Duchenne. Dr. Dongsheng Duan, a longtime researcher in Duchenne and a member of PPMD’s Scientific Advisory Committee, leads the project.
Cardiomyopathy is one of the two leading causes of morbidity and mortality in Duchenne. Gene therapy holds promise to treat Duchenne. However, current gene therapy strategies cannot fully meet the need of the heart. In adults with chronic heart failure, research shows that adeno-associated virus (AAV) SERCA2a mitigates heart failure in humans. Dr. Duan’s team found AAV SERCA2a gene therapy significantly reduced muscle disease in mdx mice thanks in part to a 2012 grant from PPMD. Now, Dr. Duan will explore if this therapy can be translated to the canine Duchenne model. The study will open the door to a new National Institutes of Health (NIH) R01 application to thoroughly evaluate AAV SERCA2a therapy for Duchenne.
“Dr. Duan is one of the leading researchers in the Duchenne space and PPMD has proudly supported the work he and his team at the University of Missouri have been doing for years. His SERCA2A work with mdx mice produced incredible results and we are excited to see this same therapy translate to the canine model. We are hopeful that one day this technology will be available for people with Duchenne. If anyone can do it, Dr. Duan and his team can.”
-- Pat Furlong, PPMD’s Founding President
Dongsheng Duan is the Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the University of Missouri School of Medicine and a member of PPMD’s Scientific Advisory Committee. He appreciates PPMD’s continued support of his research and believes the canine study will produce promising results:
“I truly appreciate the support of PPMD. This funding will allow us to start testing a highly promising therapeutic strategy for Duchenne muscular dystrophy. The results from this exploratory project will provide critical data for future studies to investigate the potential of this exciting gene therapy.”
-- Dr. Dongsheng Duan, University of Missouri
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