A particular protein may be the key to preventing Duchenne muscular dystrophy (DMD)-related heart disease — the leading cause of death in patients living with the disease — according to Rutgers University scientists.
In their study, published in the Journal of Clinical Investigation (JCI), the team examined the role of Connexin-43 (Cx43), a protein that regulates heart function. The researchers found that Cx43 was dysfunctional in both human and mouse DMD hearts, so they modified the Cx43 protein in the hopes of alleviating heart disease.
Altering the Cx43 protein through a process called phosphorylation protected DMD mice against irregular heartbeat and late-stage failure, the researchers suggest, according to a media release from Rutgers University.
“For many DMD patients, the heart muscles gradually break down, leading to death. Our findings may help give hope to millions of patients,” says study co-author Diego Fraidenraich, an assistant professor of Cell Biology and Molecular Medicine at Rutgers New Jersey Medical School.
“Medical advances have managed to slow down the disease progression in most muscles in the body, but there are yet to be any discoveries that target or prevent deterioration of the DMD heart, which remains the number one killer among these patients,” adds co-author Eric Himelman, a PhD candidate at Rutgers New Jersey Medical School.
“Therapies based on our finding may help prolong the lives of muscular dystrophy and other heart disease patients.”
Next steps include developing drugs that directly target Cx43 in DMD hearts, with a goal of potentially introducing clinical trials using Cx43 modification as a therapy for DMD patients, the researchers conclude.
[Source(s): Rutgers University, EurekAlert]
