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Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths Forward.

作者信息

Choi Soon H, Engelhardt John F

机构信息

Department of Anatomy and Cell Biology, University of Iowa, Carver College of Medicine, Iowa City, IA 52242, USA.

Department of Anatomy and Cell Biology, University of Iowa, Carver College of Medicine, Iowa City, IA 52242, USA.

出版信息

Mol Ther. 2021 Feb 3;29(2):428-430. doi: 10.1016/j.ymthe.2021.01.010. Epub 2021 Jan 12.

DOI:10.1016/j.ymthe.2021.01.010
PMID:33472034
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7854343/
Abstract
摘要

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本文引用的文献

1
Viral Vectors, Animal Models, and Cellular Targets for Gene Therapy of Cystic Fibrosis Lung Disease.病毒载体、动物模型和囊性纤维化肺病基因治疗的细胞靶标。
Hum Gene Ther. 2020 May;31(9-10):524-537. doi: 10.1089/hum.2020.013. Epub 2020 Apr 15.
2
Advances in gene therapy for cystic fibrosis lung disease.囊性纤维化肺病的基因治疗进展。
Hum Mol Genet. 2019 Oct 1;28(R1):R88-R94. doi: 10.1093/hmg/ddz139.
3
Highly Efficient Transgenesis in Ferrets Using CRISPR/Cas9-Mediated Homology-Independent Insertion at the ROSA26 Locus.利用 CRISPR/Cas9 介导的同源非依赖性插入 ROSA26 基因座在雪貂中进行高效转基因。
Sci Rep. 2019 Feb 13;9(1):1971. doi: 10.1038/s41598-018-37192-4.
4
Infection Is Not Required for Mucoinflammatory Lung Disease in CFTR-Knockout Ferrets.CFTR 基因敲除雪貂的 Mucoinflammatory 肺病不需要感染。
Am J Respir Crit Care Med. 2018 May 15;197(10):1308-1318. doi: 10.1164/rccm.201708-1616OC.
5
Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers.使用合成启动子和串联阵列增强子优化重组腺相关病毒介导的大转基因表达
Hum Gene Ther. 2015 Jun;26(6):334-46. doi: 10.1089/hum.2015.001. Epub 2015 Apr 20.
6
Lung phenotype of juvenile and adult cystic fibrosis transmembrane conductance regulator-knockout ferrets.幼年和成年囊性纤维化跨膜电导调节因子敲除雪貂的肺表型。
Am J Respir Cell Mol Biol. 2014 Mar;50(3):502-12. doi: 10.1165/rcmb.2013-0261OC.
7
New animal models of cystic fibrosis: what are they teaching us?囊性纤维化的新型动物模型：它们在向我们传授什么？
Curr Opin Pulm Med. 2011 Nov;17(6):478-83. doi: 10.1097/MCP.0b013e32834b14c9.
8
Cystic fibrosis pigs develop lung disease and exhibit defective bacterial eradication at birth.囊性纤维化猪会患上肺部疾病，并在出生时表现出细菌清除缺陷。
Sci Transl Med. 2010 Apr 28;2(29):29ra31. doi: 10.1126/scitranslmed.3000928.
9
Directed evolution of adeno-associated virus to an infectious respiratory virus.腺相关病毒向传染性呼吸道病毒的定向进化。
Proc Natl Acad Sci U S A. 2009 Mar 10;106(10):3865-70. doi: 10.1073/pnas.0813365106. Epub 2009 Feb 23.
10
Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial.重复雾化腺相关病毒载体-囊性纤维化跨膜传导调节因子治疗囊性纤维化：一项随机安慰剂对照2B期试验
Hum Gene Ther. 2007 Aug;18(8):726-32. doi: 10.1089/hum.2007.022.