Splicing defects and CRISPR-Cas9 correction in isogenic homozygous photoreceptor precursors harboring clustered deep-intronic ABCA4 variants
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Author:
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De Angeli, Pietro; Flores-Tufino, Arturo; Stingl, Katarina; Kuehlewein, Laura; Roschi, Eleonora; Wissinger, Bernd; Kohl, Susanne
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Tübinger Autor(en):
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Published in:
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Molecular Therapy Nucleic Acids
(2024), Bd.
35,
H.
1,
S.
1-12
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Verlagsangabe:
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Cambridge : Cell Press
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Language:
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English
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Full text:
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http://dx.doi.org/10.1016/j.omtn.2023.102113
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ISSN:
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2162-2531
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DDC Classifikation:
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610 - Medicine and health
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Dokumentart:
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Article
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