’Painting’ technique successfully transfers gene therapy to heart

In experiments with pigs, scientists at Johns Hopkins have successfully used a technique called “gene painting” to target gene therapy to a specific region of the heart and change the heart’s rhythm.

“Getting the genes where we want them has been a key limiting factor in the successful development of gene therapies for heart conditions,” said cardiac electrophysiologist Kevin Donahue, M.D., an associate professor at The Johns Hopkins University School of Medicine and its Heart Institute. “This new technique brings us one step closer to making gene therapies that can be tested on specific heart problems.”

The technique, if future studies in pigs and in humans are promising, could help in the development and delivery of future gene therapies for atrial fibrillation, a common ailment in which the electrical signaling that triggers the heartbeat goes awry.

Donahue led a controlled study that evaluated whether a gene therapy, using a gene called HERG-G628S that helps regulate the heartbeat, could effectively alter the heartbeat in 20 pigs with an irregular heart rhythm. The gene therapy was contained in a plastic, gel-like substance that was “painted” onto the surface of the right atrium of the heart. The gel also contained a dye so that its spread could be tracked inside the organ. After three weeks, the heartbeat had returned to normal, and the dye had penetrated only the atria.

Targeted Modification of Atrial Electrophysiology by Homogeneous Transmural Atrial Gene Transfer. Kan Kikuchi, Tetsuo Sasano, Amy McDonald, Kevin Mills and Kevin Donahue.