High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia
Saffari, Afshin, Brechmann, Barbara, Böger, Cedric, Saber, Wardiya Afshar, Jumo, Hellen, Whye, Dosh, Wood, Delaney, Wahlster, Lara, Alecu, Julian E., Ziegler, Marvin, Scheffold, Marlene, Winden, Kellen, Hubbs, Jed, Buttermore, Elizabeth D., Barrett, Lee, Borner, Georg H. H., Davies, Alexandra K., Ebrahimi-Fakhari, Darius, Sahin, Mustafa
Published in Nature communications (17.01.2024)
Published in Nature communications (17.01.2024)
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Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47
Wiseman, Jessica P, Scarrott, Joseph M, Alves-Cruzeiro, João, Saffari, Afshin, Böger, Cedric, Karyka, Evangelia, Dawes, Emily, Davies, Alexandra K, Marchi, Paolo M, Graves, Emily, Fernandes, Fiona, Yang, Zih-Liang, Coldicott, Ian, Hirst, Jennifer, Webster, Christopher P, Highley, J Robin, Hackett, Neil, Angyal, Adrienn, Silva, Thushan de, Higginbottom, Adrian, Shaw, Pamela J, Ferraiuolo, Laura, Ebrahimi-Fakhari, Darius, Azzouz, Mimoun
Published in EMBO molecular medicine (02.10.2024)
Published in EMBO molecular medicine (02.10.2024)
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