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

Alpha‑1 antitrypsin deficiency (AATM) is a congenital metabolic disorder caused by a genetic defect that leads to the incorrect production of the protective protein alpha‑1 antitrypsin (AAT). The deficiency primarily damages the lungs and the liver, and can result in serious conditions such as pulmonary emphysema, liver fibrosis, or cirrhosis. In particularly severe cases, an organ transplant is the last resort. Currently, no curative treatment is available for this disorder.