Effective splicing restoration of a deep-intronic ABCA4 variant in cone photoreceptor precursor cells by CRISPR/SpCas9 approaches

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Authors: Pietro De Angeli, Peggy Reuter, Stefan Hauser, Ludger Schöls, Katarina Sting, Bernd Wissinger, Susanne Kohl

Journal title: Molecular Therapy. Nucleic acids

Journal number: Volume 29

Journal publisher: Nature Publishing Group

Published year: 2022

Published pages: 511-524

DOI identifier: 10.1016/j.omtn.2022.07.023

ISSN: 2162-2531