ArticleDeep-intronic ABCA4 variants explain missing heritability in Stargardt disease and allow correction of splice defects by antisense oligonucleotides
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Keywords
ABCA4
antisense oligonucleotide
deep-intronic variant
missing heritability
Stargardt disease
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© 2019, The Author(s), under exclusive licence to the American College of Medical Genetics and Genomics