Detailed Phenotyping and Therapeutic Strategies for Intronic ABCA4 Variants in Stargardt Disease

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Authors: Mubeen Khan, Gavin Arno, Ana Fakin, David A. Parfitt, Patty P.A. Dhooge, Silvia Albert, Nathalie M. Bax, Lonneke Duijkers, Michael Niblock, Kwan L. Hau, Edward Bloch, Elena R. Schiff, Davide Piccolo, Michael C. Hogden, Carel B. Hoyng, Andrew R. Webster, Frans P.M. Cremers, Michael E. Cheetham, Alejandro Garanto, Rob W.J. Collin

Journal title: Molecular Therapy - Nucleic Acids

Journal number: 21

Journal publisher: Nature Publishing Group

Published year: 2020

Published pages: 412-427

DOI identifier: 10.1016/j.omtn.2020.06.007

ISSN: 2162-2531