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。 - Suppr

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超能文献

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.

作者信息

Crystal R G, Jaffe A, Brody S, Mastrangeli A, McElvaney N G, Rosenfeld M, Chu C S, Danel C, Hay J, Eissa T

机构信息

Rockefeller University Hospital, New York, New York 10021, USA.

出版信息

Hum Gene Ther. 1995 May;6(5):643-66. doi: 10.1089/hum.1995.6.5-643.

DOI:10.1089/hum.1995.6.5-643

PMID:7578401

Abstract

摘要

相似文献

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 repeat administration of a replication deficient, recombinant adenovirus containing the normal cystic fibrosis transmembrane conductance regulator cDNA to the airways of individuals with cystic fibrosis.对携带正常囊性纤维化跨膜传导调节因子cDNA的复制缺陷型重组腺病毒重复给药至囊性纤维化患者气道的评估。

Hum Gene Ther. 1995 May;6(5):667-703. doi: 10.1089/hum.1995.6.5-667.

Gene transfer to freshly isolated human respiratory epithelial cells in vitro using a replication-deficient adenovirus containing the human cystic fibrosis transmembrane conductance regulator cDNA.使用携带人囊性纤维化跨膜传导调节因子cDNA的复制缺陷型腺病毒，在体外将基因转移至新鲜分离的人呼吸道上皮细胞。

Hum Gene Ther. 1994 Mar;5(3):331-42. doi: 10.1089/hum.1994.5.3-331.

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.

[Aerosol administration of a replication defective recombinant adenovirus expressing normal human cDNA-CFTR in the respiratory tractus in patients with cystic fibrosis].[在囊性纤维化患者的呼吸道中雾化给予表达正常人cDNA-CFTR的复制缺陷型重组腺病毒]

C R Seances Soc Biol Fil. 1996;190(1):109-42.

Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector.通过局部给予正常CFTR cDNA腺病毒基因转移载体对囊性纤维化个体鼻上皮电位差的改变。

Hum Gene Ther. 1995 Nov;6(11):1487-96. doi: 10.1089/hum.1995.6.11-1487.

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 double-blind, placebo controlled, dose ranging study to evaluate the safety and biological efficacy of the lipid-DNA complex GR213487B in the nasal epithelium of adult patients with cystic fibrosis.一项双盲、安慰剂对照、剂量范围研究，旨在评估脂质-DNA复合物GR213487B在成年囊性纤维化患者鼻上皮中的安全性和生物学疗效。

Hum Gene Ther. 1998 Jan 20;9(2):249-69. doi: 10.1089/hum.1998.9.2-249.

In vivo adenovirus-mediated transfer of human CFTR cDNA to rhesus monkey airway epithelium: efficacy, toxicity and safety.体内腺病毒介导的人囊性纤维化跨膜传导调节因子（CFTR）cDNA转移至恒河猴气道上皮：疗效、毒性和安全性

Gene Ther. 1994 Nov;1(6):385-94.

Gene therapy for the respiratory manifestations of cystic fibrosis.针对囊性纤维化呼吸表现的基因治疗。

Am J Respir Crit Care Med. 1995 Mar;151(3 Pt 2):S75-87. doi: 10.1164/ajrccm/151.3_Pt_2.S75.

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Acta Naturae. 2023 Apr-Jun;15(2):20-31. doi: 10.32607/actanaturae.11708.

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.

Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome.遗传性肺部疾病和模拟婴儿猝死综合征的基因治疗：细胞和基因治疗。

Hum Gene Ther. 2012 Jun;23(6):548-56. doi: 10.1089/hum.2012.087.

L'inflammation dans la Mucoviscidose.囊性纤维化中的炎症。

Mediators Inflamm. 1996;5(2):144-69. doi: 10.1155/S0962935196000221.

Inflammation in cystic fibrosis.囊性纤维化中的炎症

Mediators Inflamm. 1996;5(2):121-43. doi: 10.1155/S096293519600021X.

Adenovirus type 37 uses sialic acid as a cellular receptor.37型腺病毒将唾液酸用作细胞受体。

J Virol. 2000 Jan;74(1):42-8.

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase.在编码人酸性α-葡萄糖苷酶的修饰腺病毒载体进行肝靶向治疗后，对II型糖原贮积病这一肌肉疾病进行全身性纠正。

Proc Natl Acad Sci U S A. 1999 Aug 3;96(16):8861-6. doi: 10.1073/pnas.96.16.8861.

Prospects for gene therapy in cystic fibrosis.囊性纤维化的基因治疗前景。

Arch Dis Child. 1999 Mar;80(3):286-9. doi: 10.1136/adc.80.3.286.

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