Novel Gene Therapy for Failing Hearts

The study was published in the February 26, 2002, issue of Circulation.

The phospholamban protein regulates the activity of a molecular pump that controls the flow of calcium in cells that might be reduced in failing hearts. Abnormal calcium levels in heart-muscle cells are often the cause of heart failure. In the study, researchers took cells from the hearts of nine end-stage heart patients who were about to have heart transplantations. Injecting single-strand DNA, which binds to the RNA message encoded by the phospholamban gene, into cultured cells prevented the formation of the phospholamban protein.

"Usually with gene therapy, you add things, but here the strategy was to take something away,” said Dr. Roger Hajjar, from the Heart Failure Center at Massachusetts General Hospital (Boston, USA), and the principal investigator. "When we targeted phospholamban, we were able to diminish it, and then calcium movement in the cells became normal.”