ADMINISTRATION OF NORMAL CDNA ADENOVIRUS VECTOR IN CYSTIC FIBROSIS

本方案将评估重组腺病毒载体AdgvCFTR重复给药的安全性和生物学有效性。10到囊性纤维化个体的气道上皮。本研究的关键要素是：将载体施用于气道的更局部区域，更仔细地定义正常CFTR cDNA表达的药效学，重复施用后评估这种表达，以及在表达盒中使用更活跃的启动子/增强子。进度报告和调查结果摘要：研究的A、C部分已经完成。共有14名患者入组。10名患者接受了三次AdGVCFTR治疗。10向量;3名患者接受了两次AdGVCFTR治疗。10向量;一名患者接受了一次AdGVCFTR治疗。10向量。在B部分，预计将有5人登记，其中2人已完成研究，每人接受了4次病媒治疗，1人目前正在登记。病人继续作为门诊病人接受随访。

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.