Novel VCP activator reverses multisystem proteinopathy nuclear proteostasis defects and enhances TDP-43 aggregate clearance

Pathogenic variants in VCP cause multisystem proteinopathy (MSP), a disease characterized by multiple clinical phenotypes including inclusion body myopathy, Paget’s disease of the bone, and frontotemporal dementia (FTD). How such diverse phenotypes are driven by pathogenic VCP variants is not known....

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Detalles Bibliográficos
Autores principales: Phan, Jessica M., Creekmore, Benjamin C., Nguyen, Aivi T., Bershadskaya, Darya D., Darwich, Nabil F., Lee, Edward B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10055171/
https://www.ncbi.nlm.nih.gov/pubmed/36993559
http://dx.doi.org/10.1101/2023.03.15.532082

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10055171/
https://www.ncbi.nlm.nih.gov/pubmed/36993559
http://dx.doi.org/10.1101/2023.03.15.532082

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