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

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

Author: De Angeli, Pietro; Reuter, Peggy; Hauser, Stefan; Schoels, Ludger; Stingl, Katarina; Wissinger, Bernd; Kohl, Susanne
Tübinger Autor(en):
De Angeli, Pietro
Reuter, Peggy
Hauser, Stefan
Stingl, Katarina
Wissinger, Bernd
Kohl, Susanne
Schöls, Ludger
Published in: Molecular Therapy - Nucleic Acids (2022), Bd. 29, S. 511-524
Verlagsangabe: Cell Press
Language: English
Full text: http://dx.doi.org/10.1016/j.omtn.2022.07.023
ISSN: 2162-2531
DDC Classifikation: 610 - Medicine and health
Dokumentart: Article
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