Professor of Pediatrics, Molecular Genetics, and Microbiology
College of Medicine
As pediatrics chair and medical director of Shands Children’s Hospital, Terence Flotte is ensuring the best medical minds continue to serve Florida’s children.
The professor of pediatrics and molecular genetics and microbiology is also a pioneer in human gene therapy. Flotte’s research has shaped one of his important departmental goals: to create a new division of cellular and molecular therapy.
“One of the key features of an academic department is the ability to add to the body of new knowledge and to use that knowledge to directly benefit patients suffering with diseases that currently do not have adequate therapy,” Flotte says. “One of the most direct ways to do that is to perform gene transfer to replace genes that have been identified as the cause of specific diseases, such as cystic fibrosis. The emergence of safe gene transfer technology has, for the first time, made it possible to test this concept in a way that ultimately could provide a new treatment for a previously incurable disease.”
Flotte currently is investigating the use of gene therapy for genetic diseases that affect children, mainly cystic fibrosis, a disorder that causes thick mucus to block the breathing passages of the lungs. Patients with the disease are predisposed to chronic lung infections.
“We discovered that when you change the virus into a vector – alter the virus and splice in the normal gene that you want to deliver – you actually change the behavior of the virus,” Flotte says. “It still sticks with the cell for a long time if that cell lasts a long time, but it doesn’t insert its DNA into the chromosome as a natural virus does. It doesn’t ‘integrate’ anymore.”
Flotte says that while meticulous care by health professionals has resulted in longer lives for many patients with the disease, gene therapy may be key to finding a cure.