Detailed Phenotyping and Therapeutic Strategies for Intronic ABCA4 Variants in Stargardt Disease.
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 Molecular therapy Nucleic acids（2020）
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