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: | , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2022-09-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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