Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy

Abstract

Efforts to develop gene therapies for cystic fibrosis (CF) helped to drive vector innovations more than two decades ago, including the first use of recombinant adenovirus (rAd) and recombinant adeno-associated virus (rAAV) for in vivo gene therapy and for human use. These early-generation vectors failed to progress in clinical trials, however, because of immune responses and the failure of these episomal vectors to result in stable transduction of the airway progenitor cells, particularly basal cells in the conducting airways. Two articles in the current issue of Human Gene Therapy address this issue directly by using newer vector systems: helper-dependent adenovirus (HD-Ad) and lentivirus (LV) vectors.