Parent Project Muscular Dystrophy awards $280,000 for cardiac gene therapy

Yesterday, we announced that the University of Missouri’s Dongsheng Duan, Ph.D. was awarded a grant of $280,000 by PPMD to continue his work with SERCA2a.

We all know how important a healthy heart is, especially in Duchenne. And because you thought with your heart and cheered on Olympic hero Ryan Lochte by giving to the Go for the Gold campaign this summer, we were able to continue our support of Dr. Duan’s important work. So thank you…Together, we went for the gold and made it happen!

About the project

The heart is a muscle too. This is something we stress over and over again in our fight to end Duchenne. Talented researchers like Dr. Duan understand that the best therapies in the world will do patients no good if their hearts can't handle the therapy. Therefore, this research will give us the building blocks we need to eventually create wholly successful therapeutics that patients can withstand.

Dr. Duan and his team at the University of Missouri want to determine if delivering the gene for the calcium-handling protein SERCA2a is sufficient to correct cardiac disease in mdx mice or if the dystrophin gene must be delivered as well. This is important information since SERCA2a gene therapy is in human testing now for non-Duchenne related cardiomyopathy.

Heart failure is a leading cause of death in people with advanced Duchenne. Through the course of the disease, heart function is progressively compromised by calcium overload and inadequate transport, which leads to cell imbalance, dysfunction, and death. SERCA2a is a calcium ion pump that may be able to reduce the damage caused by calcium overload.

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