Browsing Bergen Open Research Archive by Author "Gaspert, Ariana"

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Systems analyses of the Fabry kidney transcriptome and its response to enzyme replacement therapy identified and cross-validated enzyme replacement therapy-resistant targets amenable to drug repurposing

Delaleu, Nicolas; Marti, Hans Peter; Strauss, Philipp; Sekulic, Miroslav; Osman, Tarig Al-Hadi; Tøndel, Camilla; Skrunes, Rannveig; Leh, Sabine; Svarstad, Einar; Nowak, Albina; Gaspert, Ariana; Rusu, Elena; Kwee, Ivo; Rinaldi, Andrea; Flatberg, Arnar; Eikrem, Øystein Solberg (Journal article; Peer reviewed, 2023)

Fabry disease is a rare disorder caused by variations in the alpha-galactosidase gene. To a degree, Fabry disease is manageable via enzyme replacement therapy (ERT). By understanding the molecular basis of Fabry nephropathy ...