Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B

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Authors: Juliane W Schott, Peixin Huang, Michael Morgan, Jennifer Nelson-Brantley, Ally Koehler, Bryan Renslo, Hildegard Büning, Athanasia Warnecke, Axel Schambach, Hinrich Staecker

Journal title: Molecular Therapy

Journal publisher: Cell Press

Published year: 2023

DOI identifier: 10.1016/j.ymthe.2023.10.018

ISSN: 1525-0024