Article
Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2017
Borel Florie, Tang Qiushi, Gernoux Gwladys, Greer Cynthia, Wang Ziqiong, Barzel Adi, Kay Mark A, Shultz Leonard D, Greiner Dale L, Flotte Terence R, Brehm Michael A, Mueller Christian
Abstract excerpt
Hepatocytes represent an important target for gene therapy and editing of single-gene disorders. In α-1 antitrypsin (AAT) deficiency, one missense mutation results in impaired secretion of AAT. In most patients, lung damage occurs due to a lack of AAT-mediated protection of lung elastin from neutrophil elastase. In some patients, accumulation of misfolded PiZ mutant AAT protein triggers hepatocyte injury, leading...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.