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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10541187/
https://www.ncbi.nlm.nih.gov/pubmed/37561581
http://dx.doi.org/10.1172/JCI171356

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