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

Tuesday, August 11, 2009 - 05:35 in Health & Medicine

Gene therapy researchers 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. Three patients, apparently for the first time in their lives, produced trace amounts of the protective form of a protein called alpha-1 antitrypsin for up to one year, a potential step toward a gene therapy for about 100,000 Americans with alpha-1 antitrypsin deficiency.

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