Gene therapy trial succeeds in spurring production of a protective protein

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. "University of Massachusetts Medical School":[ http://www.umassmed.edu/Content.aspx?id=77592]

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