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Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: a phase I trial of safety and efficacy in the nasal airway.

作者信息

Sorscher E J, Logan J J, Frizzell R A, Lyrene R K, Bebok Z, Dong J Y, Duvall M D, Felgner P L, Matalon S, Walker L

出版信息

Hum Gene Ther. 1994 Oct;5(10):1259-77. doi: 10.1089/hum.1994.5.10-1259.

DOI:10.1089/hum.1994.5.10-1259
PMID:7531503
Abstract
摘要

相似文献

1
Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: a phase I trial of safety and efficacy in the nasal airway.
Hum Gene Ther. 1994 Oct;5(10):1259-77. doi: 10.1089/hum.1994.5.10-1259.
2
Adenovirus-mediated gene transfer for cystic fibrosis: Part A. Safety of dose and repeat administration in the nasal epithelium. Part B. Clinical efficacy in the maxillary sinus.腺病毒介导的基因转移治疗囊性纤维化：A部分。鼻上皮中剂量及重复给药的安全性。B部分。上颌窦的临床疗效。
Hum Gene Ther. 1995 Feb;6(2):205-18. doi: 10.1089/hum.1995.6.2-205.
3
Cystic fibrosis gene therapy using an adenovirus vector: in vivo safety and efficacy in nasal epithelium.使用腺病毒载体的囊性纤维化基因治疗：鼻上皮的体内安全性和有效性
Hum Gene Ther. 1994 Feb;5(2):209-19. doi: 10.1089/hum.1994.5.2-209.
4
A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial.雾化脂质体介导的基因治疗囊性纤维化的I/IIa期安全性和有效性研究支持多剂量试验。
Am J Respir Crit Care Med. 2015 Dec 1;192(11):1389-92. doi: 10.1164/rccm.201506-1193LE.
5
A phase 1 study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung.一项针对囊性纤维化患者的1期研究，旨在评估单次给予一种携带正常囊性纤维化跨膜传导调节因子基因cDNA的复制缺陷型重组腺病毒在肺部的安全性、毒性和生物学疗效。
Hum Gene Ther. 1995 May;6(5):643-66. doi: 10.1089/hum.1995.6.5-643.
6
A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.一项关于脂质体介导的基因转移至囊性纤维化患者鼻上皮的安慰剂对照研究。
Gene Ther. 1997 Mar;4(3):199-209. doi: 10.1038/sj.gt.3300391.
7
Gene therapy for cystic fibrosis using E1-deleted adenovirus: a phase I trial in the nasal cavity. The University of North Carolina at Chapel Hill.使用E1缺失腺病毒治疗囊性纤维化的基因疗法：鼻腔内的I期试验。北卡罗来纳大学教堂山分校。
Hum Gene Ther. 1994 May;5(5):615-39. doi: 10.1089/hum.1994.5.5-615.
8
Gene therapy for cystic fibrosis: which postman, which box?囊性纤维化的基因治疗：哪个邮递员，哪个信箱？
Thorax. 1998 Mar;53(3):197-9. doi: 10.1136/thx.53.3.197.
9
In vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis.体内鼻电位差：评估囊性纤维化基因转移疗效的技术与方案
Hum Gene Ther. 1995 Apr;6(4):445-55. doi: 10.1089/hum.1995.6.4-445.
10
Cystic fibrosis. Vehicles for gene therapy.囊性纤维化。基因治疗载体。
Nature. 1993 Oct 21;365(6448):691-2. doi: 10.1038/365691a0.

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The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways.羧甲基纤维素凝胶在增加小鼠气道中非病毒基因转导中的应用。
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Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing.通过AAV6.2介导的片段反式剪接在囊性纤维化气道上皮细胞中实现功能性囊性纤维化跨膜传导调节因子表达。
Hum Gene Ther. 2009 Mar;20(3):267-81. doi: 10.1089/hum.2008.173.
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Lipoplex-mediated delivery of nucleic acids: factors affecting in vivo transfection.脂质体介导的核酸递送：影响体内转染的因素
J Mol Med (Berl). 2004 Sep;82(9):579-91. doi: 10.1007/s00109-004-0558-8. Epub 2004 Jun 23.
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Pathol Oncol Res. 1999;5(1):3-15. doi: 10.1053/paor.1999.0003.