Gene Therapy Shows Promise for Heart Failure

Researchers from the University of Michigan and University of Minnesota successfully used gene transfer therapy to improve the function of cardiomyopathic hearts. The belief is that this kind of “closed heart surgery” may one day prove effective as a clinical therapy.

To make this advance, Herron [Todd J. Herron, Ph.D., research assistant professor of molecular and integrative physiology at the University of Michigan –ed.] and colleagues treated heart muscle cells from the failing hearts of rabbits and humans with a virus (adenovirus) modified to carry a gene which produces a protein that enables heart cells to contract normally (fast molecular motor) or a gene that becomes active in failing hearts, which is believed to be part of the body’s way of coping with its perilous situation (slow molecular motor). Heart cells treated with the gene to express the fast molecular motor contracted better, while those treated with the gene to express the slow molecular motor were unaffected.
"Helping hearts heal themselves, rather than prescribing yet another drug to sustain a failing organ, would be a major advance for doctors and patients alike," said Gerald Weissmann, M.D., editor-in-chief of the FASEB Journal. "Equally important, it shows that gene therapy remains one of the most promising approaches to treating the world’s most common and deadliest diseases."
Current clinical agents and treatments focus on the amount of calcium available for contraction, which can provide short-term cardiac benefits, but are associated with an increased mortality in the long-term. Results from this study show that calcium-independent treatments could have implications for heart diseases associated with depressed heart function, due to the effectiveness of fast molecular motor gene transfer on the improved contractions of human heart muscle cells.

Abstract in FASEB Journal: Ca²⁺-independent positive molecular inotropy for failing rabbit and human cardiac muscle by {alpha}-myosin motor gene transfer
University of Michigan: Scientists jump-start heart cells by gene transfer…