Tag: <span>antitrypsin</span>

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New gene editing approach for alpha-1 antitrypsin deficiency shows promise

A new study by scientists at UMass Medical School shows that using a technique called “nuclease-free” gene editing to correct cells with the mutation that causes a rare liver disease leads to repopulation of the diseased liver with healthy cells. Alpha-1 antitrypsin deficiency is an inherited disease that causes liver and lung damage; the Mueller...