Promising breakthrough in Cystic Fibrosis research: Nebulization of CFTR gene therapy
For the first time, a non-viral gene therapy in a phase 2 trial has been demonstrated to be advantageous to lung function in people afflicted with cystic fibrosis (1). Normally, people with cystic fibrosis have one or more mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations … Continue reading