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Rapamycin and Chloroquine: The In Vitro and In Vivo Effects of Autophagy-Modifying Drugs Show Promising Results in Valosin Containing Protein Multisystem Proteinopathy

Mutations in the valosin containing protein (VCP) gene cause hereditary Inclusion body myopathy (hIBM) associated with Paget disease of bone (PDB), frontotemporal dementia (FTD), more recently termed multisystem proteinopathy (MSP). Affected individuals exhibit scapular winging and die from progress...

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Detalles Bibliográficos
Autores principales: Nalbandian, Angèle, Llewellyn, Katrina J., Nguyen, Christopher, Yazdi, Puya G., Kimonis, Virginia E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4401571/
https://www.ncbi.nlm.nih.gov/pubmed/25884947
http://dx.doi.org/10.1371/journal.pone.0122888