Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing-Reference-Cited by-同舟云学术

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Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

Published:2023-03 Issue: Volume:31 Page:16-27
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Jo Dong Hyun,Jang Hyeon-Ki,Cho Chang Sik,Han Jun Hee,Ryu Gahee,Jung Youngri,Bae Sangsu,Kim Jeong Hun^ORCID

Funder

National Research Foundation of Korea

Korea Drug Development Fund

Seoul National University Hospital

Korea Research Institute of Bioscience and Biotechnology

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference53 articles.

1. Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy;Cideciyan;Prog. Retin. Eye Res.,2010

2. Spotlight on childhood blindness;Sahel;J. Clin. Invest.,2011

3. Leber congenital amaurosis (LCA): potential for improvement of vision;Cideciyan;Invest. Ophthalmol. Vis. Sci.,2019

4. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial;Russell;Lancet,2017

5. Gene therapy and genome surgery in the retina;DiCarlo;J. Clin. Invest.,2018

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