Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype.

doi:10.15252/emmm.202013742

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Article

Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype.

EMBO molecular medicine - 7 Oct 2021

Soldati Chiara, Lopez-Fabuel Irene, Wanderlingh Luca G, Garcia-Macia Marina, Monfregola Jlenia, Esposito Alessandra, Napolitano Gennaro, Guevara-Ferrer Marta, Scotto Rosato Anna, Krogsaeter Einar K, Paquet Dominik, Grimm Christian M, Montefusco Sandro, Braulke Thomas, Storch Stephan, Mole Sara E, De Matteis Maria A, Ballabio Andrea, Sampaio Julio L, McKay Tristan, Johannes Ludger, Bolaños Juan P, Medina Diego L

Abstract excerpt

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-conjugated bacterial toxins to label Gb3 to develop a cell-based high content imaging (HCI) screening assay for the repurposing of...

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Identifiers

PMID: 34411438
DOI: 10.15252/emmm.202013742
PMCID: PMC8495452

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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