GENE THERAPY FOR THE RESPIRATORY MECHANISMS OF CYSTIC FIBROSIS--SAFETY AND EFF

囊性纤维化呼吸机制的基因治疗——安全性和有效性

• 批准号: 3741600
• 负责人: RONALD CRYSTAL
• 金额: --
• 依托单位: ROCKEFELLER UNIVERSITY
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3741600
• 关键词: Adenoviridae; chloride channels; complementary DNA; cystic fibrosis; gene therapy; human subject; human therapy evaluation; recombinant virus; respiratory epithelium; transfection /expression vector

This study is designed to evaluate the hypothesis that repetitive administration of a replication deficient recombinant adenovirus vector containing the human CFTR cDNA to the airway epithelium of individuals with cystic fibrosis will safely correct the biologic abnormalities of the airway epithelium of CF on a chronic basis.

这项研究旨在评估重复性的假设， 施用复制缺陷型重组腺病毒载体 将含有人CFTR cDNA的重组质粒转染至个体的气道上皮， 囊性纤维化将安全地纠正生物学异常， 慢性CF的气道上皮。