QR-1011 restores defective ABCA4 splicing caused by multiple severe ABCA4 variants underlying Stargardt disease

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Authors: Kaltak, M.; de Bruijn, Petra; van Leeuwen, Willemijn; Platenburg, G.; Cremers, F.P.M.; Collin, R.W.J.; Swildens, J.

Journal title: Scientific Reports

Journal number: 14:684

Journal publisher: Nature Publishing Group

Published year: 2024

DOI identifier: 10.1038/s41598-024-51203-7

ISSN: 2045-2322