Gene therapy for cystic fibrosis in a mouse model using adeno-associated viral vectors KU Leuven
Cystic fibrosis (CF) is the most common monogenic life-threatening disease in the Caucasian population, caused by mutations in CFTR, a chloride/bicarbonate channel that regulates fluid transport across epithelium of different organs (airways, pancreas, intestine, sweat glands and vas deferens). CF affects multiple organs, but lung pathology is the major clinical manifestation. Mutations in the CFTR gene lead to an imbalance ...