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。 - Suppr

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.

作者信息

Welsh M J, Zabner J, Graham S M, Smith A E, Moscicki R, Wadsworth S

机构信息

Howard Hughes Medical Institute, Department of Internal Medicine, University of Iowa College of Medicine, Iowa City 52242, USA.

出版信息

Hum Gene Ther. 1995 Feb;6(2):205-18. doi: 10.1089/hum.1995.6.2-205.

DOI:10.1089/hum.1995.6.2-205

PMID:7537540

Abstract

摘要

相似文献

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.

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.

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.

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.

Evaluation of the efficacy and safety of in vitro, adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA.体外腺病毒介导的人囊性纤维化跨膜传导调节因子cDNA转移的疗效和安全性评估。

Hum Gene Ther. 1994 Jun;5(6):717-29. doi: 10.1089/hum.1994.5.6-717.

Gene therapy for cystic fibrosis utilizing a replication deficient recombinant adenovirus vector to deliver the human cystic fibrosis transmembrane conductance regulator cDNA to the airways. A phase I study.

Hum Gene Ther. 1994 Aug;5(8):1019-57. doi: 10.1089/hum.1994.5.8-1019.

Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis.向囊性纤维化患者的鼻上皮重复施用编码囊性纤维化跨膜传导调节因子的腺病毒载体。

J Clin Invest. 1996 Mar 15;97(6):1504-11. doi: 10.1172/JCI118573.

In vivo evaluation of the safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lung.腺病毒介导的人囊性纤维化跨膜传导调节因子cDNA转导至肺的安全性的体内评估。

Hum Gene Ther. 1994 Jun;5(6):731-44. doi: 10.1089/hum.1994.5.6-731.

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.

Gene therapy of cystic fibrosis lung disease using E1 deleted adenoviruses: a phase I trial.使用E1缺失腺病毒对囊性纤维化肺病进行基因治疗：一项I期试验。

Hum Gene Ther. 1994 Apr;5(4):501-19. doi: 10.1089/hum.1994.5.4-501.

引用本文的文献

Genome Editing for Cystic Fibrosis.基因组编辑治疗囊性纤维化。

Cells. 2023 Jun 6;12(12):1555. doi: 10.3390/cells12121555.

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.

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.

Cystic Fibrosis Gene Therapy: Looking Back, Looking Forward.囊性纤维化基因治疗：回顾与展望。

Genes (Basel). 2018 Nov 7;9(11):538. doi: 10.3390/genes9110538.

Apical localization of the coxsackie-adenovirus receptor by glycosyl-phosphatidylinositol modification is sufficient for adenovirus-mediated gene transfer through the apical surface of human airway epithelia.通过糖基磷脂酰肌醇修饰实现柯萨奇病毒-腺病毒受体的顶端定位足以介导腺病毒通过人气道上皮细胞的顶端表面进行基因转移。

J Virol. 2001 Aug;75(16):7703-11. doi: 10.1128/JVI.75.16.7703-7711.2001.

A chimeric type 2 adenovirus vector with a type 17 fiber enhances gene transfer to human airway epithelia.一种带有17型纤维的嵌合2型腺病毒载体可增强基因向人呼吸道上皮细胞的转移。

J Virol. 1999 Oct;73(10):8689-95. doi: 10.1128/JVI.73.10.8689-8695.1999.

Potential use of T cell receptor genes to modify hematopoietic stem cells for the gene therapy of cancer.T细胞受体基因在修饰造血干细胞用于癌症基因治疗方面的潜在应用。

Pathol Oncol Res. 1999;5(1):3-15. doi: 10.1053/paor.1999.0003.

Lack of high affinity fiber receptor activity explains the resistance of ciliated airway epithelia to adenovirus infection.缺乏高亲和力纤维受体活性解释了纤毛气道上皮对腺病毒感染的抗性。

J Clin Invest. 1997 Sep 1;100(5):1144-9. doi: 10.1172/JCI119625.

Generation of cytotoxic T lymphocytes against immunorecessive epitopes after multiple immunizations with adenovirus vectors is dependent on haplotype.用腺病毒载体多次免疫后，针对免疫隐性表位产生细胞毒性T淋巴细胞取决于单倍型。

J Virol. 1997 Mar;71(3):2277-84. doi: 10.1128/JVI.71.3.2277-2284.1997.

Adenovirus-mediated gene transfer to ciliated airway epithelia requires prolonged incubation time.腺病毒介导的基因转移至纤毛气道上皮需要较长的孵育时间。

J Virol. 1996 Oct;70(10):6994-7003. doi: 10.1128/JVI.70.10.6994-7003.1996.

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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.

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