Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype

Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype

Abstract Batten diseases (BDs) are a group of lysosomal storage disorders characterized by seizure, visual loss, and cognitive and motor deterioration. We discovered increased levels of globotriaosylceramide (Gb3) in cellular and murine models of CLN3 and CLN7 diseases and used fluorescent‐conjugate...

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Main Authors: Chiara Soldati, Irene Lopez‐Fabuel, Luca G Wanderlingh, Marina Garcia‐Macia, Jlenia Monfregola, Alessandra Esposito, Gennaro Napolitano, Marta Guevara‐Ferrer, Anna Scotto Rosato, Einar K Krogsaeter, Dominik Paquet, Christian M Grimm, Sandro Montefusco, Thomas Braulke, Stephan Storch, Sara E Mole, Maria A De Matteis, Andrea Ballabio, Julio L Sampaio, Tristan McKay, Ludger Johannes, Juan P Bolaños, Diego L Medina
Format: Article
Language:English
Published: Springer Nature 2021-08-01
Series:EMBO Molecular Medicine
Subjects:
CLN3
CLN7
high content imaging screening
tamoxifen
TFEB
Online Access:https://doi.org/10.15252/emmm.202013742
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