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Gene Therapy Trial Succeeds In Boosting Protective Protein In Patients With Hereditary Lung Disease

Researchers at the University of Massachusetts Medical School and the University of Florida in Gainesville have safely given new, functional genes to patients with a hereditary defect that can lead to fatal lung and liver diseases, according to clinical trial findings slated to appear this week in the online early edition of the Proceedings of the National Academy of Science.

"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 Terence R. Flotte, MD, dean of the School of Medicine and provost & executive deputy chancellor of UMass Medical School. Dr. Flotte, senior author on the study, was formerly the chair of pediatrics at the University of Florida, where the study was conducted.

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