Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation

Title
Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation
Authors
Keywords
antisense oligonucleotides, genetic therapy, splice redirection, USH2A, Usher syndrome
Journal
Molecular Therapy-Nucleic Acids
Volume 5, Issue -, Pages e381
Publisher
Elsevier BV
Online
2016-11-02
DOI
10.1038/mtna.2016.89

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