Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy
We are pleased to announce that a new study, with contributions by several MERI members, has just been published in The Journal of Clinical Investigation! The paper, entitled ‘Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy’, provides a preclinical validation of targeted ion channel functional rescue, reinforcing the effectiveness of nonviral genome editing therapy for rare inherited disorders.
Keywords: Gene editing, nanoparticles, nonviral delivery, KCNJ13 loss-of-function nonsense mutation, 61 Kir7.1 ion channel, Leber congenital amaurosis 16 (LCA16), inherited retinal degeneration, induced 62 pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE)
