Selected Grantee Publications

Prime Editing–Mediated Correction of the CFTR W1282X Mutation in iPSCs and Derived Airway Epithelial Cells

Li et al., PLOS ONE. 2023.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10686454/

Cystic fibrosis (CF) is caused by recessive mutations in the CF transmembrane conductance regulator (CFTR) gene. Correction of nonsense CFTR mutations, which affects 10% of CF patients, via genomic editing represents a promising therapeutic approach. In this study, investigators tested whether prime editing can be applied as a potential therapeutic modality. Induced pluripotent stem cells (iPSCs) from a CF patient homozygous for the CFTR W1282X mutation were used. Studies demonstrated that prime editing corrected mutant allele in iPSCs, which effectively restored CFTR function in iPSC-derived airway epithelial cells and organoids. Supported by ORIP (R01OD01026594).