Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.
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DOI: 10.1016/j.jhep.2018.03.012
Abstract: BACKGROUND & AIMS Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder causing pulmonary and liver disease. The PiZ mutation in AAT (SERPINA1) results in mis-folded AAT protein (Z-AAT) accumulating in hepatocytes, leading to fibrosis and… read more here.
Keywords: production; aat; alpha antitrypsin; arc aat ... See more keywords