New research from Dr. Michael Rudnicki’s team firmly establishes the important role muscle stem cells play in Duchenne muscular dystrophy (DMD). People with this inherited disease develop severe muscle weakness and can no longer walk by age 12.
The team previously discovered that DMD affects muscle stem cells as well as muscle fibers. In this study published in the Journal of Cachexia, Sarcopenia and Muscle, Marie Esper and Dr. Caroline Brun looked at what was happening in the muscles of mouse models of DMD from birth to adulthood.
Their findings confirmed what Dr. Rudnicki’s team had seen in previous experiments – that muscle stem cells in DMD are not producing enough of the cells that eventually become muscle cells. Muscle repair is consequently inefficient, and this is responsible for aggressive disease progression in DMD. A therapy that corrects this problem is now being tested in clinical trials led by Satellos Bioscience, a startup co-founded by Dr. Rudnicki.
“This study confirms that we’ll need to target both muscle fibres and muscle stem cells in order to treat this disease,” said Dr. Rudnicki, senior scientist at The Ottawa Hospital, professor at the University of Ottawa and Chief Discovery Officer at Satellos.
Authors: Marie E. Esper, Caroline E. Brun, Alexander Y. T. Lin, Peter Feige, Marie J. Catenacci, Marie-Claude Sincennes, Morten Ritso, Michael A. Rudnicki
Funding: US National Institutes for Health, Canadian Institutes for Health Research, the Muscular Dystrophy Association, Defeat Duchenne Canada, the European Joint Programme on Rare Diseases, Canadian Institutes of Health Research, Muscular Dystrophy Canada, Stem Cell Network.
Core Resources: StemCore Laboratories, Preclinical Imaging Core, Cell Biology and Image Acquisition Core
The Ottawa Hospital is a leading academic health, research and learning hospital proudly affiliated with the University of Ottawa. All researchers at The Ottawa Hospital follow a Responsible Innovation Framework for developing and commercializing innovations in a responsible way.
