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BACKGROUND & AIMS
In most genetic diseases, the goal of gene therapy is to deliver a particular transgene; however, sometimes a… Expand alpha1-Antitrypsin (alpha1-AT) is the most abundant circulating proteinase inhibitor. The Z variant results in profound plasma… Expand OBJECTIVES
To ascertain how long 120 mg/kg alpha1-antitrypsin concentrate (alpha1-AT-C), administered I.V. every 2 weeks, can… Expand We have theorized that a subset of PiZZ α1-antitrypsin (α1-AT)-deficient individuals is more susceptible to liver injury by… Expand