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Article

Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy.

The Journal of clinical investigation - 2 Oct 2023

Kabra Meha, Shahi Pawan K, Wang Yuyuan, Sinha Divya, Spillane Allison, Newby Gregory A, Saxena Shivani, Tong Yao, Chang Yu, Abdeen Amr A, Edwards Kimberly L, Theisen Cole O, Liu David R, Gamm David M, Gong Shaoqin, Saha Krishanu, Pattnaik Bikash R

Abstract excerpt

Clinical genome editing is emerging for rare disease treatment, but one of the major limitations is the targeting of CRISPR editors' delivery. We delivered base editors to the retinal pigmented epithelium (RPE) in the mouse eye using silica nanocapsules (SNCs) as a treatment for retinal degeneration. Leber congenital amaurosis type 16 (LCA16) is a rare pediatric blindness caused by point mutations in the KCNJ13...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 37561581
DOI: 10.1172/JCI171356
PMCID: PMC10541187

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