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Hereditary lung and liver disease successfully treated for the first time using gene correction
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.
AI Summary
Alpha‑1 antitrypsin deficiency (AATM) is a congenital metabolic disorder caused by a genetic defect that produces an incorrectly folded alpha‑1 antitrypsin protein. The defective protein damages the lungs and liver, leading to conditions such as pulmonary emphysema, liver fibrosis, and cirrhosis. In severe cases, organ transplantation is the only remaining option. Currently, no curative treatment exists for AATM.
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