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Ottawa scientists transform natural protein into possible muscle regeneration drug

November 29, 2013

Dr. Michael Rudnicki and his colleagues have modified a natural protein that promotes muscle regeneration, making it much easier to manufacture and deliver to people affected by diseases such as Duchenne muscular dystrophy. The results, published in the prestigious journal Nature Communications, pave the way for human clinical trials.

“There has been worldwide interest in using this family of proteins to enhance organ and tissue regeneration, but we’ve been stuck at the laboratory stage because these proteins are difficult to manufacture and difficult to deliver to tissues within the body,” explained lead author Dr. Michael Rudnicki of the Ottawa Hospital Research Institute and the University of Ottawa. “Now we’ve found that a small part of the protein has just as much regenerative power as the whole thing, but it is much easier to manufacture and it can spread throughout tissues much more easily. This is a real breakthrough that could lead to the development of new therapies for devastating diseases that affect the muscles, and possibly other organs.”

The protein in question, called Wnt7a, is part of a large protein family that plays a key role in stimulating stem cells and promoting tissue regeneration. Fate Therapeutics, Inc., a public biopharmaceutical company co-founded by Dr. Rudnicki, is currently advancing Wnt7a-based protein analogs in preclinical development and plans to initiate human clinical trials within a few years. These therapeutics could be helpful in treating patients with genetic diseases, such as Duchenne muscular dystrophy, as well as muscle weakness associated with cancer and other diseases.

Dr. Rudnicki is a senior scientist, director of the Regenerative Medicine Program and director of the Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute. He is also a professor at the University of Ottawa and the scientific director of Canada’s Stem Cell Network and he holds a Canada Research Chair in Molecular Genetics. Dr. Rudnicki recently received the Dr. J. David Grimes Career Achievement Award from the Ottawa Hospital Research Institute.

This research was funded by the Muscular Dystrophy Association, the Canadian Institutes of Health Research, the National Institutes of Health, the Ontario Ministry of Research and Innovation, the Canada Research Chair Program and The Ottawa Hospital Foundation.

Full reference: A truncated Wnt7a retains full biological activity in skeletal muscle. Julia von Maltzahn, Radoslav Zinoviev, Natasha C. Chang, C. Florian Bentzinger & Michael A. Rudnicki. Nature Communications. November 29, 2013. DOI: 10.1038/ncomms3869.

About the Ottawa Hospital Research Institute
The Ottawa Hospital Research Institute (OHRI) is the research arm of The Ottawa Hospital and is an affiliated institute of the University of Ottawa, closely associated with the university’s Faculties of Medicine and Health Sciences. OHRI includes more than 1,700 scientists, clinical investigators, graduate students, postdoctoral fellows and staff conducting research to improve the understanding, prevention, diagnosis and treatment of human disease. www.ohri.ca

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Ottawa Hospital Research Institute
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