Adenine base editing is an efficient approach to restore function in FA patient cells without double-stranded DNA breaks
Sebastian M. Siegner, Laura Ugalde, Alexandra Clemens, Laura García‐García, Juan Antonio Bueren, Paula Rı́o, Mehmet E. Karasu, Jacob E. Corn
This study demonstrates that adenine base editing can restore FANCA function in FA patient cells without causing DNA breaks, showing promise for therapeutic applications.
Nature Communications · Nov 12, 2022Peer-reviewedMolecular Biology