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NEW YORK (GenomeWeb) – Arrowhead Research today announced that it has begun dosing in a Phase I trial of its siRNA-based alpha-1 antitrypsin (AAT) deficiency therapy ARC-AAT.

AAT deficiency is a genetic disease that leads to decreased activity of the protease inhibitor alpha-1 antitrypsin in the blood and lungs causing lung dysfunction. It can also feature the accumulation of a mutant AAT protein known as Z-AAT in liver tissue, which leads to liver injury, fibrosis, cirrhosis, and potentially liver cancer. ARC-AAT is designed to lower production of Z-AAT.

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