Splicing defects and CRISPR-Cas9 correction in isogenic homozygous photoreceptor precursors harboring clustered deep-intronic ABCA4 variants

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Authors: Pietro De Angeli, Arturo Flores-Tufiño, Katarina Stingl, Laura Kühlewein, Eleonora Roschi, Bernd Wissinger, Susanne Kohl

Journal title: Molecular Therapy Nucleic Acids

Journal number: 35(1)102113

Journal publisher: Nature Publishing Group

Published year: 2023

DOI identifier: 10.1016/j.omtn.2023.102113

ISSN: 2162-2531