H5.001CBCFTR

H5.001CBCFTR is a third-generation, replication-deficient adenoviral gene therapy vector designed for the treatment of cystic fibrosis. This 'gutless' or helper-dependent vector is derived from adenovirus serotype 5 (Ad5) and has been engineered with deletions in the E1, E2, and E4 regions to minimize viral gene expression, thereby reducing the host's inflammatory and immune response and potentially extending the duration of the transgene expression. The vector carries a cDNA expression cassette for the human cystic fibrosis transmembrane conductance regulator (CFTR) gene, typically driven by a cytomegalovirus (CMV) or a hybrid promoter. It is administered endobronchially to deliver functional CFTR genes to the respiratory epithelium, aiming to restore chloride ion transport and improve pulmonary function in patients.