Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations
Kalyan Dulla, Ralph W. N. Slijkerman, Hester C. van Diepen, Sílvia Albert, Margo Dona, Wouter Beumer, Janne J. Turunen, Hee Lam Chan, Iris A. E. Schulkens, Lars Vorthoren, Cathaline den Besten, Levi C.M. Buil, Iris Schmidt, Jiayi Miao, Hanka Venselaar, , 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.
Molecular Therapy · Apr 23, 2021Peer-reviewedGenetics