URMC receives $3M to advance research on ARDS, gene therapy

May. 23, 2016

David Dean, Ph.D., professor of Pediatrics and Neonatology at the University of Rochester Medical Center, has received a $3 million grant to explore a novel method of gene therapy delivery that could greatly benefit patients with acute respiratory distress syndrome (ARDS).

The syndrome, which occurs after trauma such as an injury or pneumonia, causes alveoli (tiny air sacs in the lungs) to take on too much water and prevents the lungs from efficiently processing oxygen. It affects about 150,000 people each year, and there is no cure — about 40 percent of people who are diagnosed die as a result.

Dean’s approach attempts to influence the affected alveoli from the inside out by using gene therapy to communicate with the cells’ nuclei and decision-making centers. Using a carefully-devised DNA compound, the treatment compels lung cells to release their extra water, allowing them to function properly again.

However, our cells are well-equipped to prevent rogue strands of DNA from entering – great for keeping viruses out, but challenging for researchers who are trying to get helpful compounds in. Using a process called electroporation, Dean delivers a measured shock of electricity that causes the cells in a target area to briefly open, allowing DNA inside.

Electroporation is a burgeoning science in the field of gene therapy and there is minimal literature on its use across the chest and lungs. The grant, from the National Institutes of Health, will allow Dean to continue to test its safety and efficacy.

“Our research has generated very encouraging results thus far,” said Dean. “If it continues to show promise, our next step will be a clinical trial.”

The grant, which began earlier this year, supports four years of research.