Effective splicing restoration of a deep-intronic ABCA4 variant in cone photoreceptor precursor cells by CRISPR/SpCas9 approaches
Author:
Funder
HORIZON EUROPE Marie Sklodowska-Curie Actions
European Commission
Horizon 2020 Framework Programme
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference65 articles.
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4. The gene for Stargardt disease, ABCA4, is a major retinal gene: a mini-review;Koenekoop;Ophthalmic genetic,2003
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1. EDSpliCE, a CRISPR-Cas9 gene editing platform to rescue splicing, effectively corrects inherited retinal dystrophy-associated splicing defects;2024-03-30
2. Splicing defects and CRISPR-Cas9 correction in isogenic homozygous photoreceptor precursors harboring clustered deep-intronic ABCA4 variants;Molecular Therapy - Nucleic Acids;2024-03
3. ABCA4 c.6480-35A>G, a novel branchpoint variant associated with Stargardt disease;Frontiers in Genetics;2023-09-07
4. Advances towards personalized therapies for Stargardt disease;Expert Review of Ophthalmology;2023-09-03
5. Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function;Journal of Translational Medicine;2023-08-16
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