Ad5-CB-CFTR

Ad5-CB-CFTR is an experimental first-generation (E1-deleted), replication-deficient adenoviral gene therapy based on serotype 5 (Ad5). It was developed to treat cystic fibrosis by delivering a functional copy of the human cystic fibrosis transmembrane conductance regulator (CFTR) cDNA to the respiratory epithelium. The expression of the transgene is driven by a chicken beta-actin (CB) promoter, often in combination with a CMV enhancer. The primary goal of the therapy is to restore the production of the CFTR protein on the apical surface of airway cells, thereby correcting the defective chloride ion transport and sodium hyperabsorption that characterize the disease. Clinical development, primarily led by the University of Iowa in collaboration with Genzyme, focused on evaluating the safety and efficacy of nasal and pulmonary delivery. Although early trials demonstrated successful gene transfer and partial restoration of chloride channel function, the program was limited by the transient nature of gene expression and host inflammatory responses to the adenoviral vector.

Other names

Ad5-CFTRAd-5-CFTRAd 5-CFTRAd5-CB-hCFTRAd-5-CB-hCFTRAd 5-CB-hCFTRE1-deleted adenovirus-CFTRE-1-deleted adenovirus-CFTRE 1-deleted adenovirus-CFTR