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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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Bibliographische Detailangaben
Veröffentlicht in:PLoS One
Hauptverfasser: Nalbandian, Angèle, Llewellyn, Katrina J., Nguyen, Christopher, Yazdi, Puya G., Kimonis, Virginia E.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Public Library of Science 2015
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4401571/
https://ncbi.nlm.nih.gov/pubmed/25884947
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0122888
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