Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C

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Authors: Melita Kaltak, Petra de Bruijn, Davide Piccolo, Sang-Eun Lee, Kalyan Dulla, Thomas Hoogenboezem, Wouter Beumer, Andrew R. Webster, Rob W.J. Collin, Michael E. Cheetham, Gerard Platenburg, Jim Swildens

Journal title: Molecular Therapy Nucleic Acids

Journal number: 31

Journal publisher: Nature Publishing Group

Published year: 2023

Published pages: 674-688

DOI identifier: 10.1016/j.omtn.2023.02.020

ISSN: 2162-2531