Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disease that results from mutations in the alpha-1 antitrypsin (
Shuling Guo, Sheri L. Booten, Mariam Aghajan, Gene Hung, Chenguang Zhao, Keith Blomenkamp, Danielle Gattis, Andrew Watt, Susan M. Freier, Jeffery H. Teckman, Michael L. McCaleb, Brett P. Monia
Robust AAT reduction in cynomolgus hepatocytes and in vivo after AAT-ASO treatment.