Nov 18, 2015 11:36 AM EST
Defective Stem Cells: Duchenne Muscular Dystrophy May Be Caused by Dystrophin Lack in Stem Cells, Stars Support Duchenne Charity

Duchenne muscular dystrophy is a degenerative genetic condition that affects one in 3,600 children. An individual with this disease loses vital body functions over time and may die by their mid 20s to early 30s.  The common causes of death in these cases are weakened, even scarred, heart and lung muscles.

In the past, experts attributed muscular degeneration in Duchenne muscular dystrophy to flawed muscle fibres.  A recent study, however, found that the fault occurs even further back.

The study's senior author, and Ottawa Hospital Research Institute director of regenerative medicine, Dr. Michael Rudnicki says, "For nearly 20 years, we've thought that the muscle weakness observed in patients with Duchenne muscular dystrophy is primarily due to problems in their muscle fibres, but our research shows that it is also due to intrinsic defects in the function of their muscle stem cells."

"This completely changes our understanding of Duchenne muscular dystrophy and could eventually lead to far more effective treatments."

The research team's findings suggest that the protein dystrophin, which is responsible for the repair of muscle fibres from injuries and daily wear-and-tear, is not only present in the muscle fibres but also in the muscle stem cells. Stem cells are undifferentiated cells from which rise precursor or blast cells. Fibres are generated from these cells to build and repair muscles in the body. In Duchenne muscular dystrophy, dystrophin is either lacking or insufficient in the stem cells.  Without dystrophin, stem cells produce ten times less of the precursor cells for muscles.

Dr. Rudnick and his team are hopeful, "I think that down the road, it's possible we will be able to repair those deficiencies of the stem cells using drugs."

"If we can repair the stem cell deficit ... that may well significantly ameliorate the disorder."

Interest in Duchenne muscular dystrophy has been increasing, especially on the aspect of research for cure and treatment. Cure Duchenne, by Paul and Debra Miller, and Action Duchenne are among the organisations actively supporting research to eliminate Duchenne muscular dystrophy. Cure Duchenne has received support from such celebrities as Justin Bieber, Seal, Sofia Vergara, Wolfgang Puck, Mandy Moore, Clay Matthews, David Foster and Ryan Getzlaf.

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