Search Results - "Böger, Cedric"

High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia by 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

“…Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect therapeutic targets for rare diseases. In this study, we developed…”
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Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47 by 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

“…Spastic paraplegia 47 (SPG47) is a neurological disorder caused by mutations in the adaptor protein complex 4 β1 subunit ( AP4B1 ) gene leading to AP-4 complex…”
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