Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation Resistant Human α1-antitrypsin for the Treatment of α1-antitrypsin Deficiency.
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DOI: 10.1089/hum.2022.192
Abstract: α1-antitrypsin (AAT) deficiency is a common autosomal recessive hereditary disorder, with a high risk for the development of early onset panacinar emphysema. AAT produced primarily in the liver, functions to protect the lung from neutrophil… read more here.
Keywords: aav8haat avl; antitrypsin; intravenous administration; study ... See more keywords