Editas Medicine has developed a therapy named, EDIT-101, which delivers SaCas9 directly to remove the intronic IVS26 mutation in the CEP20 gene, implicated in aberrant splicing, thereby restoring functional CEP20 levels in human cells and humanized CEP20 mice.77 A clinical trial of EDIT-101 by Allergan and Editas Medicine paves the way for a prospective curative strategy for treating congenital blindness using an in vivo approach. Here, CEP20 is linked to congenital stationary night blindness.