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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

1
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。
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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。
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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。
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[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]
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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.
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Advances in gene therapy for cystic fibrosis lung disease.囊性纤维化肺病的基因治疗进展。
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Hum Gene Ther. 2012 Jun;23(6):548-56. doi: 10.1089/hum.2012.087.
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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.
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Inflammation in cystic fibrosis.囊性纤维化中的炎症
Mediators Inflamm. 1996;5(2):121-43. doi: 10.1155/S096293519600021X.
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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。
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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。
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Arch Dis Child. 1999 Mar;80(3):286-9. doi: 10.1136/adc.80.3.286.