Hepatic-targeted RNA interference provides persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.

Alice M Turner,Jan Stolk,Robert Bals,Jason D Lickliter,James Hamilton,Dawn R Christianson,Bruce D Given,Jonathan G Burdon,Rohit Loomba,James K Stoller,Jeffery H Teckman

Journal of Hepatology（2018）

引用 68|浏览4

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摘要

•Alpha-1 antitrypsin deficiency is associated with liver fibrosis and cirrhosis.•RNAi drugs might benefit patients with alpha-1 antitrypsin deficiency.•ARC-AAT reduced alpha-1 antitrypsin protein in study participants.

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关键词

siRNA,Phase 1,PiZZ,RNAi,Therapeutic

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