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

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

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...

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Bibliographic Details
Main Authors: Meha Kabra, Pawan K. Shahi, Yuyuan Wang, Divya Sinha, Allison Spillane, Gregory A. Newby, Shivani Saxena, Yao Tong, Yu Chang, Amr A. Abdeen, Kimberly L. Edwards, Cole O. Theisen, David R. Liu, David M. Gamm, Shaoqin Gong, Krishanu Saha, Bikash R. Pattnaik
Format: Article
Language:English
Published: American Society for Clinical Investigation 2023-10-01
Series:The Journal of Clinical Investigation
Subjects:
Ophthalmology
Online Access:https://doi.org/10.1172/JCI171356

Internet

https://doi.org/10.1172/JCI171356

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