Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47

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Authors: Jessica P Wiseman, Joseph M Scarrott, João Alves-Cruzeiro, Afshin Saffari, Cedric Böger, Evangelia Karyka, Emily Dawes, Alexandra K Davies, Paolo M Marchi, Emily Graves, Fiona Fernandes, Zih-Liang Yang, Ian Coldicott, Jennifer Hirst, Christopher P Webster, J Robin Highley, Neil Hackett, Adrienn Angyal, Thushan de Silva, Adrian Higginbottom, Pamela J Shaw, Laura Ferraiuolo, Darius Ebrahimi-Fakhar

Journal title: EMBO Molecular Medicine

Journal number: 16

Journal publisher: EMBO PRESS, GERMANY

Published year: 2024

Published pages: 2882-2917

DOI identifier: 10.1038/s44321-024-00148-5

ISSN: 1757-4684