Lentiviral vectors for gene therapy of cystic fibrosis.

摘要: A replication-defective vector based on human immunodeficiency virus (HIV) was evaluated for gene transfer directed to the lung. The tropism of this has been expanded through incorporation vesticular stomatitis G protein into its envelope. HIV effectively transduced nondividing airway epithelial cells in vitro whereas a murine-based retroviral did not. Experiments bronchial xenograft model demonstrated high-level transduction with cystic fibrosis transmembrane conductance regulator (CFTR) undifferentiated, (CF)-derived xenograft. CFTR expression stable and capable functional correction CF defect after graft matured. not transduce when instilled epithelium had differentiated. This block appears be at level entry, although post entry restrictions cannot ruled out. Further development system therapy should focus better understanding potential blocks.