The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease

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Lysosomal storage diseases (LSDs) are inherited disorders caused by lysosomal deficiencies and characterized by dysfunction of the autophagy-lysosomal pathway (ALP) often associated with neurodegeneration. No cure is currently available to treat neuropathology in LSDs. By studying a mouse model of m...

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Detalles Bibliográficos
Autores principales: Monaco, Antonio, Maffia, Veronica, Sorrentino, Nicolina Cristina, Sambri, Irene, Ezhova, Yulia, Giuliano, Teresa, Cacace, Vincenzo, Nusco, Edoardo, De Risi, Maria, De Leonibus, Elvira, Schrader, Thomas, Klärner, Frank-Gerrit, Bitan, Gal, Fraldi, Alessandro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7132627/
https://www.ncbi.nlm.nih.gov/pubmed/32087148
http://dx.doi.org/10.1016/j.ymthe.2020.02.005
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