GeneticsMolecular TherapyPeer-reviewed
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations
Kalyan Dulla, Ralph W. N. Slijkerman, Hester C. van Diepen…·Erwin van Wijk
This study demonstrates that QR-421a induces exon 13 skipping in USH2A transcripts, restoring retinal function in zebrafish and non-human primates.
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