Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency

Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency

Abstract Multiple sulfatase deficiency (MSD, MIM #272200) results from pathogenic variants in the SUMF1 gene that impair proper function of the formylglycine‐generating enzyme (FGE). FGE is essential for the posttranslational activation of cellular sulfatases. MSD patients display reduced or absent...

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Main Authors: Lars Schlotawa, Karolina Tyka, Matthias Kettwig, Rebecca C Ahrens‐Nicklas, Matthias Baud, Tea Berulava, Nicola Brunetti‐Pierri, Alyssa Gagne, Zackary M Herbst, Jean A Maguire, Jlenia Monfregola, Tonatiuh Pena, Karthikeyan Radhakrishnan, Sophie Schröder, Elisa A Waxman, Andrea Ballabio, Thomas Dierks, André Fischer, Deborah L French, Michael H Gelb, Jutta Gärtner
Format: Article
Language:English
Published: Springer Nature 2023-02-01
Series:EMBO Molecular Medicine
Subjects:
drug screening
formylglycine‐generating enzyme
lysosomal disorder
retinoids
sulfatase‐modifying factor 1
Online Access:https://doi.org/10.15252/emmm.202114837
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