Genome scale analysis of pathogenic variants targetable for single base editing.
Alexander V LavrovGeorgi G VarenikovMikhail Yu SkoblovPublished in: BMC medical genomics (2020)
Protein engineering helps to develop new enzymes with a narrower window of base editors as well as using new Cas9 enzymes with different PAM sequences. But, even now the list of mutations which can be targeted with currently available systems is huge enough to choose and develop new targeted therapies.