Antisense Oligonucleotide-Based Rescue of Aberrant Splicing Defects Caused by 15 Pathogenic Variants in ABCA4

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Authors: Tomasz Z. Tomkiewicz,Nuria Suárez-Herrera,Frans P. M. Cremers, Rob W. J. Collin and Alejandro Garanto

Journal title: International Journal of Molecular Sciences

Journal number: 22(9):4621

Journal publisher: Multidisciplinary Digital Publishing Institute (MDPI)

Published year: 2021

Published pages: 1-17

DOI identifier: 10.3390/ijms22094621

ISSN: 1422-0067