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

A study published in the journal Molecular Therapy by Christian Mueller, PhD, shows that using nuclease-free gene editing to correct cells with the mutation that causes alpha-1 antitrypsin deficiency leads to repopulation of a diseased liver with healthy cells. It has the potential to prevent liver and lung damage from forming in very young alpha-1 patients.