Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4.

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Authors: Nuria Suárez-Herrera, Iris B. Riswick, Irene Vázquez-Domínguez, Lonneke Duijkers, Dyah W. Karjosukarso, Davide Piccolo, Miriam Bauwens, Elfride De Baere, Michael E. Cheetham, Alejandro Garanto, and Rob W.J. Collin.

Journal title: Molecular Therapy

Journal number: vol. 32, no. 3

Journal publisher: Nature Publishing Group

Published year: 2024

DOI identifier: 10.1016/j.ymthe.2024.01.019

ISSN: 1525-0016