Article

Nonviral base editing of <i>KCNJ13</i> mutation preserves vision in an inherited retinal channelopathy

2022-07-13

Abstract excerpt

Clinical genome editing is emerging for rare disease treatment, but one of the major limitations is the targeted delivery of CRISPR editors. We delivered base editors to the retinal pigmented epithelium (RPE) in the mouse eye using silica nanocapsules (SNC) as a treatment for retinal degeneration. Leber Congenital Amaurosis (LCA16) is a rare pediatric blindness caused by point mutations in the KCNJ13 gene, a loss...

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Literature Corpus work: 4eaa1b8c-0755-5c9a-989e-8824c865ce41
DOI: 10.1101/2022.07.12.499808

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