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Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells

10 Jul 2024

Abstract excerpt

Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human cystic fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE-engineered PE guide RNAs, the...

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PMID: 38987629
DOI: 10.1038/s41551-024-01233-3

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