Parent Project Muscular Dystrophy Awards University of Missouri $50,000 Exploratory Grant to Explore SERCA2a in Duchenne Dog Study

HACKENSACK, N.J., Feb. 16, 2015 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to the fight to end Duchenne muscular dystrophy (Duchenne), announced today that they will award Dr. Dongsheng Duan and his team at the Department of Molecular Microbiology and Immunology at the University of Missouri School of Medicine, a $50,000 exploratory grant to test SERCA2a gene therapy as a potential treatment for dogs with Duchenne.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 to 5,000 live male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficult breathing, and heart failure.  Death typically occurs in the mid-to-late 20s.

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," said PPMD's Founding President Pat Furlong. "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."

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," Duan said. "The results from this exploratory project will provide critical data for future studies to investigate the potential of this exciting gene therapy."

To learn more about Parent Project Muscular Dystrophy's grant opportunities, please visit our website. To see a full list of what we are funding, click here.

About Parent Project Muscular Dystrophy                    

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest, most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

Photo - http://photos.prnewswire.com/prnh/20150213/175491
Logo - http://photos.prnewswire.com/prnh/20100119/DC39975LOGO

SOURCE Parent Project Muscular Dystrophy

Related Links

http://www.parentprojectmd.org