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

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

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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 transplant is the last resort. There is as yet no curative treatment.

A team from the Department of Gastroenterology, Hepatology, Infectious Diseases and Endocrinology and the Department of Pneumology and Infectious Diseases at Hanover Medical School (MHH), in collaboration with Renji Hospital in Shanghai, China, has now succeeded for the first time in correcting the underlying genetic defect and treating seven patients with AATM.

Using CRISPR/Cas9 technology—known as "gene scissors"—and a biotechnological tool, the researchers specifically modified a tiny defective building block in the genetic material of the AAT-producing liver cells. In technical terms, this procedure is known as base editing. The initial findings on the safety and efficacy of this gene correction have been published in the Journal of Hepatology.

Alpha-1 antitrypsin is mainly produced in the liver and travels via the blood to the lungs, where it protects the tissue from being broken down by the body's own enzymes. "Like most congenital genetic disorders, alpha-1 antitrypsin deficiency is caused by a single tiny building block in the DNA—known as a base—being incorrectly incorporated," explains Dr.


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