WS17.02 Improved adenine base editing approach to correct W1282X-CFTR

Abstract

Aim: W1282X is the sixth most common cystic fibrosis (CF)-causing variant and one of those without an approved therapy. Adenine base editing (ABE) has attracted interest as it can correct mutations without creating a double-stranded break in DNA, being thus potentially safer than the traditional approach with CRISPR/Cas9. Efficient correction of W1282X by ABE has been reported but bystander edits have been observed at the adjacent adenines.