Gene therapy to treat severe heart failure

NEW YORK, June 20 (UPI) -- U.S. scientists say they are setting up clinical trials for therapeutic gene injections that could take the place of a heart transplant.

Patients enrolled in the New York-Presbyterian Hospital/Columbia University Medical Center gene therapy clinical trial -- the Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease -- will receive a specially engineered gene that stimulates production of an enzyme necessary for the heart to pump more efficiently because of severe heart failure.

"This new therapy seeks to replenish the levels of this enzyme by introducing the gene for SERCA2a, which is depressed in these patients," principal investigator Dr. Donna Mancini said in a statement.

"If proven effective, this approach could be an alternative to heart transplant for patients without any other options."

Gene therapy is a technique for correcting defective genes responsible for disease development by inserting genes into a patient's cells and tissues. In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal" disease-causing gene.