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Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype

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 bacteri...

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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8495452/ https://www.ncbi.nlm.nih.gov/pubmed/34411438 http://dx.doi.org/10.15252/emmm.202013742

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