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A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction.一项使用小鼠模型的演示表明，对囊性纤维化进行成功的基因治疗仅需要部分基因校正。
Gene Ther. 1996 Sep;3(9):797-801.
2
Mode of formation and structural features of DNA-cationic liposome complexes used for transfection.用于转染的DNA-阳离子脂质体复合物的形成方式和结构特征。
Biochemistry. 1993 Jul 20;32(28):7143-51. doi: 10.1021/bi00079a011.
3
Nasal application of the cationic liposome DC-Chol:DOPE does not alter ion transport, lung function or bacterial growth.
Eur Respir J. 1994 Mar;7(3):442-5. doi: 10.1183/09031936.94.07030442.
4
Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium.囊性纤维化鼻上皮离子转运缺陷的体内测量方案。
Eur Respir J. 1994 Nov;7(11):2050-6.
5
Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor.通过靶向聚合免疫球蛋白受体将基因转移到动物气道上皮中。
J Clin Invest. 1995 Feb;95(2):493-502. doi: 10.1172/JCI117690.
6
Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis.腺病毒介导的基因转移可短暂纠正囊性纤维化患者鼻上皮细胞中的氯转运缺陷。
Cell. 1993 Oct 22;75(2):207-16. doi: 10.1016/0092-8674(93)80063-k.
7
Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.脂质体介导的囊性纤维化跨膜传导调节因子基因转移至囊性纤维化患者的鼻上皮细胞。
Nat Med. 1995 Jan;1(1):39-46. doi: 10.1038/nm0195-39.
8
A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis.一项关于腺病毒载体介导的基因转移在囊性纤维化患者鼻上皮中的对照研究。
N Engl J Med. 1995 Sep 28;333(13):823-31. doi: 10.1056/NEJM199509283331302.
9
Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis.将携带人囊性纤维化跨膜传导调节因子（CFTR）互补DNA（cDNA）的腺病毒施用于囊性纤维化患者的呼吸道。
Nat Genet. 1994 Sep;8(1):42-51. doi: 10.1038/ng0994-42.
10
Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis.重组腺病毒中E2a的失活改善了囊性纤维化基因治疗的前景。
Nat Genet. 1994 Jul;7(3):362-9. doi: 10.1038/ng0794-362.

囊性纤维化的基因治疗：哪个邮递员，哪个信箱？

Gene therapy for cystic fibrosis: which postman, which box?

作者信息

Middleton P G, Alton E W

机构信息

Department of Respiratory Medicine, Westmead Hospital, Australia.

出版信息

Thorax. 1998 Mar;53(3):197-9. doi: 10.1136/thx.53.3.197.

DOI:10.1136/thx.53.3.197
PMID:9659356
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC1745161/
Abstract

Since 1989 when the gene responsible for cystic fibrosis was cloned and designated the cystic fibrosis transmembrane conductance regulator (CFTR) gene, considerable progress has been made in the development of gene therapy for this disease. Clinical trials have already been performed using cationic liposome and adenoviral based gene transfer systems, measuring the safety and efficacy of this new form of treatment, with variable results to date. These two approaches and the current progress in airway gene delivery are discussed.

摘要

自1989年导致囊性纤维化的基因被克隆并命名为囊性纤维化跨膜传导调节因子（CFTR）基因以来，针对这种疾病的基因治疗取得了相当大的进展。已经使用基于阳离子脂质体和腺病毒的基因转移系统进行了临床试验，以评估这种新治疗形式的安全性和有效性，迄今为止结果不一。本文讨论了这两种方法以及气道基因递送的当前进展。