adeno-associated virus-CFTR vector

Adeno-associated virus-CFTR vector (AAV-CFTR) is a gene therapy designed to treat cystic fibrosis by delivering a functional copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The therapy typically employs a recombinant adeno-associated virus (rAAV), most commonly serotype 2 (AAV2), as a viral vector to transduce the airway epithelium. Once inside the host cells, the vector facilitates the expression of the CFTR protein, which functions as a chloride channel. This restoration of chloride transport is intended to correct the underlying biochemical defect in cystic fibrosis, improving mucus clearance and reducing lung infections. Early clinical development was spearheaded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and Target Genetics (with the specific candidate tgAAVCF). Although Phase I and II trials demonstrated the safety and feasibility of gene transfer, the program was ultimately terminated after failing to show sustained clinical efficacy in larger trials.

Other names

recombinant adeno-associated virus-CFTRAAV-CFTR vectorAAV-CFTR gene therapy