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Liver gene therapy: The magic bullet for the sick lung.

作者信息

Brunetti-Pierri Nicola

机构信息

Department of Translational Medicine, Section of Pediatrics, Federico II University, Naples, Italy.

Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.

出版信息

Mol Ther Methods Clin Dev. 2022 Jun 15;26:72-73. doi: 10.1016/j.omtm.2022.05.004. eCollection 2022 Sep 8.

Abstract
摘要

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本文引用的文献

1
Liver-directed gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice.
Mol Ther Methods Clin Dev. 2022 Apr 13;25:425-438. doi: 10.1016/j.omtm.2022.04.003. eCollection 2022 Jun 9.
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Ferret models of alpha-1 antitrypsin deficiency develop lung and liver disease.
JCI Insight. 2022 Mar 8;7(5):e143004. doi: 10.1172/jci.insight.143004.
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Alpha-1 antitrypsin deficiency: clarifying the role of the putative protective threshold.
Eur Respir J. 2022 Feb 10;59(2). doi: 10.1183/13993003.01410-2021. Print 2022 Feb.
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Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter.
Mol Ther Methods Clin Dev. 2020 Jul 9;18:620-630. doi: 10.1016/j.omtm.2020.07.005. eCollection 2020 Sep 11.
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Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.
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Editing out five paralogs to create a mouse model of genetic emphysema.
Proc Natl Acad Sci U S A. 2018 Mar 13;115(11):2788-2793. doi: 10.1073/pnas.1713689115. Epub 2018 Feb 16.
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Circulating polymers in α1-antitrypsin deficiency.
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