Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function

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Authors: Melita Kaltak; Rocio Blanco-Garavito; Laurie L. Molday; Claire-Marie Dhaenens; Eric E. Souied; Gerard Platenburg; Jim Swildens; Robert S. Molday; Frans P. M. Cremers

Journal title: Journal of Translational Medicine

Journal number: 21

Journal publisher: BioMed Central

Published year: 2023

Published pages: 546

DOI identifier: 10.1186/s12967-023-04406-x

ISSN: 1479-5876