ADMINISTRATION OF NORMAL CDNA ADENOVIRUS VECTOR IN CYSTIC FIBROSIS

该方案将评价向囊性纤维化个体的气道上皮重复施用重组腺病毒载体AdgvCFTR.10的安全性和生物学功效。 这项研究的关键要素是：管理的载体更局部的区域的气道，更仔细的定义的药效学的表达的正常CFTR cDNA，评价这种表达后重复管理，并使用一个更活跃的启动子/增强子的表达盒。进度报告及结果摘要：研究的A及C部分已完成。 共入组14例患者。 10载体的三次施用;三名患者接受了两次AdGVCFTR. 10载体的施用;并且一名患者接受了一次AdGVCFTR. 10载体的施用。 在B部分，计划入组5名个体，其中2名个体已完成研究，且均接受了4次载体给药，1名个体目前正在入组。 患者继续作为门诊患者接受随访。

This protocol will evaluate the safety and biologic efficacy of repeat administration of the recombinant adenovirus vector AdgvCFTR.10 to the airway epithelium of individuals with cystic fibrosis. Key elements of this study are: administration of the vector to more localized areas of the airways, more careful definition of the pharmacodynamics of expression of the normal CFTR cDNA, evaluation of this expression following repeat administration, and use of a more active promoter/enhancer in the expression cassette. Progress report and summary of findings: Parts A and C of the study have been completed. A total of fourteen patients were enrolled. Ten patients have received three administrations of the AdGVCFTR.10 vector; three patients have received two administrations of the AdGVCFTR.10 vector; and one patient has received one administration of the AdGVCFTR.10 vector. In part B, five individuals are projected to enroll, two of which have completed the study and have each had four administrations of the vector, one individual is currently enrolled. Patients continue to be followed as outpatients.