Prime Editing Corrects Cystic Fibrosis Mutation
Researchers have refined the primary editing (PE) method for correcting the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), a significant cause of cystic fibrosis. They combined several efficiency improvements, including engineered PE guide RNAs and PEmax architecture. They achieved high correction efficiency in immortalized bronchial epithelial cells and substantial correction in patient-derived […]