MIT Technology Review - "Doubling Down On Gene Therapy For Heart Failure" - Emily Mullin
For over a decade, scientists have been trying to reverse heart failure by delivering a new gene to the heart that makes it better at pumping blood and supplying the body with oxygen. A team at the Icahn School of Medicine at Mount Sinai, led by Roger Hajjar, MD, director of the cardiovascular research center and Arthur & Janet C. Ross professor of medicine at the Icahn School of Medicine at Mount Sinai, has just tested such a therapy in pigs, with promising results. Dr. Hajjar was a cofounder of the biotech company called Celladon, which sponsored the failed clinical trial. “By identifying molecular targets that are known across the board in all patients with heart failure, we could treat all patients instead of just patients with certain genetic mutations,” said Dr. Hajjar. The goal is to reverse heart failure with a single injection. This time around, Dr. Hajjar said, he and his team have reengineered the viral vector to make it more effective at delivering the new gene to the heart.
- Roger Hajjar, MD, Director, Cardiovascular Research Center, Arthur & Janet C. Ross Professor, Medicine, Icahn School of Medicine at Mount Sinai