GAINESVILLE, Fla., Aug. 11 (UPI) -- U.S. scientists say they've had success in a genetic therapy trial in spurring production of a protective protein in hereditary lung disease patients.
Researchers at the University of Florida and the University of Massachusetts Medical School said they gave new functional genes to patients with a hereditary defect that can lead to fatal lung and liver diseases.
"This trial represents a very important step toward a potential gene therapy for the 100,000 or more Americans who suffer with alpha-1 antitrypsin deficiency," said Dr. Terence Flotte, senior author of the study.
Patients with alpha-1 antitrypsin deficiency cannot produce a protective form of the protein alpha-1 antitrypsin, which is normally produced in the liver and protects the lungs from inflammation. Those lacking alpha-1 antitrypsin are vulnerable to infections or irritants in the air, such as cigarette smoke, and often develop life-threatening lung disease.
In the study, three patients who received injections of a harmless virus containing copies of a correct gene for alpha-1 protein were able to produce trace amounts of alpha-1 antitrypsin for up to one year. Although the levels produced were not considered therapeutic, the researchers said the study provided critical "proof of principle" that a corrected, functioning gene could trigger production of the protein.
The research appears in the online early edition of the Proceedings of the National Academy of Science.
