In vivo genome editing for inherited retinal disease: Opportunities and challenges-Reference-Cited by-同舟云学术

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In vivo genome editing for inherited retinal disease: Opportunities and challenges

Published:2024-08 Issue:8 Volume:32 Page:2433-2434
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Collin Rob W.J.^ORCID,Leroy Bart P.

Publisher

Elsevier BV

Reference14 articles.

1. Gene Editing for CEP290-Associated Retinal Degeneration;Pierce;N. Engl. J. Med.,2024

2. Leber Congenital Amaurosis (LCA): Potential for Improvement of Vision;Cideciyan;Invest. Ophthalmol. Vis. Sci.,2019

3. Outcome Measures for Clinical Trials of Leber Congenital Amaurosis Caused by the Intronic Mutation in the CEP290 Gene;Jacobson;Invest. Ophthalmol. Vis. Sci.,2017

4. Clinical Characterization of 66 Patients With Congenital Retinal Disease Due to the Deep-Intronic c.2991+1655A>G Mutation in CEP290;Valkenburg;Invest. Ophthalmol. Vis. Sci.,2018

5. Mutations in the CEP290 (NPHP6) Gene Are a Frequent Cause of Leber Congenital Amaurosis;Den Hollander;Am. J. Hum. Genet.,2006

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