Article
A helper-dependent adenoviral vector rescues CFTR to wild-type functional levels in cystic fibrosis epithelial cells harbouring class I mutations.
The European respiratory journal - 1 Nov 2020
Cao Huibi, Ouyang Hong, Laselva Onofrio, Bartlett Claire, Zhou Zhichang Peter, Duan Cathleen, Gunawardena Tarini, Avolio Julie, Bear Christine E, Gonska Tanja, Hu Jim, Moraes Theo J
Abstract excerpt
Cystic fibrosis (CF) is a genetic disorder affecting multiple organs, including the pancreas, hepatobiliary system and reproductive organs; however, lung disease is responsible for the majority of morbidity and mortality. Management of CF involves CF transmembrane conductance regulator (CFTR) modulator agents including corrector drugs to augment cellular trafficking of mutant CFTR as well as potentiators that...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
