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Hereditary lung and liver disease successfully treated for the first time using gene correction

Medical Xpress - latest medical and health news stories · 1h ago

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Alpha-1 antitrypsin deficiency (AATM) is a congenital metabolic disorder. It is caused by a genetic defect, which results in the protective protein alpha-1 antitrypsin (AAT) being produced incorrectly. This deficiency primarily damages the lungs and the liver, where it can lead to serious conditions such as pulmonary emphysema, liver fibrosis or cirrhosis. In particularly severe cases, an organ…

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