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

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

Author: De Angeli, Pietro; Flores-Tufino, Arturo; Stingl, Katarina; Kuehlewein, Laura; Roschi, Eleonora; Wissinger, Bernd; Kohl, Susanne
Tübinger Autor(en):
De Angeli, Pietro
Stingl, Katarina
Roschi, Eleonora
Wissinger, Bernd
Kohl, Susanne
Flores-Tufiño, Arturo
Kühlewein, Laura
Published in: Molecular Therapy Nucleic Acids (2024), Bd. 35, H. 1, S. 1-12
Verlagsangabe: Cambridge : Cell Press
Language: English
Full text: http://dx.doi.org/10.1016/j.omtn.2023.102113
ISSN: 2162-2531
DDC Classifikation: 610 - Medicine and health
Dokumentart: Article
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