Assessing Human Airway Epithelial Progenitor Cells for Cystic Fibrosis Cell Therapy.

ABSTRACT: Cystic fibrosis (CF) is caused by loss-of-function mutations in the CFTR (CF transmembrane regulator) gene. Pharmacologic therapies directed at CFTR have been developed but are not effective for mutations that result in little or no mRNA or protein expression. Cell therapy is a potential mutation-agnostic approach to treatment. One strategy is to harvest human bronchial epithelial cells (HBECs) for gene addition or genetic correction, followed by expansion and engraftment. This approach will require cells to grow extensively while retaining their ability to reconstitute CFTR activity. We hypothesized that conditionally reprogrammed cell (CRC) technology, namely growth in the presence of irradiated feeder cells and a Rho kinase inhibitor, would enable expansion while maintaining cell

SUBMITTER: Lee RE 

PROVIDER: S-EPMC7462339 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature