Liver-disease mutation corrected in human stem cells

A TEAM of researchers has corrected a faulty gene in induced pluripotent stem (iPS) cells derived from skin cells of people with an inherited metabolic liver disease. The researchers then developed the stem cells into something resembling liver cells. Their work was published in Nature.'1-antitrypsin deficiency (A1ATD) is the most common genetic disease of the liver, and is caused by a single nucleotide change in the gene that codes for '1-antitrypsin (A1AT), an enzyme inhibitor that normally protects bodily tissues. The condition can result in cirrhosis of liver, which can be treated only with a liver transplant, and in an increased risk of lung cancer and emphysema.
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