Antisense oligonucleotide- and CRISPR-Cas9-mediated rescue of mRNA splicing for a deep intronic CLRN1 mutation

Published 2020 View Full Article

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Title
Antisense oligonucleotide- and CRISPR-Cas9-mediated rescue of mRNA splicing for a deep intronic CLRN1 mutation
Authors
Keywords
CLRN1, mRNA splicing, antisense oligonucleotide, AON, CRISPR-Cas9, Usher syndrome, USH, USH3, splicing mutation, gene editing
Journal
Molecular Therapy-Nucleic Acids
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-07-31
DOI
10.1016/j.omtn.2020.07.036

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