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

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

Stargardt disease is an autosomal recessively inherited retinal disorder commonly caused by pathogenic variants in the ABCA4 gene encoding the ATP-binding cassette subfamily A member 4 (ABCA4) protein. Several deep-intronic variants in ABCA4 have been classified as disease causing. By strengthening...

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Main Authors: Pietro De Angeli (Author), Peggy Reuter (Author), Stefan Hauser (Author), Ludger Schöls (Author), Katarina Stingl (Author), Bernd Wissinger (Author), Susanne Kohl (Author)
Format: Book
Published: Elsevier, 2022-09-01T00:00:00Z.
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3rd Floor Main Library

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