DNA-PKcs Inhibition Improves Sequential Gene Insertion of the Full-Length CFTR cDNA in Airway Stem Cells.

A preprint abstract from biorxiv.org discusses the potential use of autologous airway stem cell therapies for treating cystic fibrosis (CF). The study explores the use of small molecules AZD7648 and ART558 to improve the insertion of the CFTR cDNA into human airway basal stem cells. The addition of AZD7648 alone improved gene insertion by 2-3 times, while the combination of ART558 and AZD7648 further improved gene insertion but caused toxicity. The study suggests that further research is needed to validate the effects of AZD7648 treatment on cells. [Extracted from the article]