Saving eyesight, one gene at a time.
Trends in molecular medicine, 29(7): 484-486 PMID 37169661 DOI 10.1016/j.molmed.2023.04.008
Abstract
Kai Yao's group used prime editing to repair a blindness-causing mutation in the Pde6b gene in the mouse retina. This breakthrough was made possible by a Cas9 nickase that is not constrained by a protospacer adjacent motif (PAM) sequence requirement. This innovation brings prime editing technology one step closer to correcting disease-causing mutations at will.