In DMD, the most common form of muscular dystrophy, patients lack a large, rod-like protein called dystrophin located primarily in muscles used for movement and in heart muscle. The dystrophin is part of a group of proteins that acts as an anchor, connecting each muscle cell's structural framework with the lattice of proteins and other molecules outside the cell. Without dystrophin, many of the muscle cells in the heart are damaged, subsequently die and are replaced by connective tissue.
“Many Duchenne MD patients suffer from dilated cardiomyopathy (DCM), a condition in which the chambers of the heart are enlarged and weakened,” explained the study’s lead author, Suzanne Berry, Ph.D. “As a result the heart can’t efficiently pump blood to the body and many patients eventually die. We hypothesized that mesoangioblast stem cells (ADM) found in the walls of large blood vessels, in this case the aorta, would restore dystrophin and therefore alleviate or prevent DCM.”