C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy
Year: 2020
Type: article
Abstract: -targeted therapeutics reduce TDP-43 pathology in vivo, and demonstrate that alterations in plasma NFL provide insight into the therapeutic efficacy of disease-modifying treatments.
Source: Science Translational Medicine
Authors: Casey N. Cook, Yanwei Wu, Hana M. Odeh, Tania F. Gendron, Karen Jansen-West, Giulia del Rosso, Mei Yue, Peizhou Jiang, Edward Gomes, Jimei Tong, Lillian M. Daughrity, Nicole M. Avendano, Monica Castanedes-Casey, Wei Shao, Björn Oskarsson, Giulio S. Tomassy, Alexander McCampbell, Frank Rigo, Dennis W. Dickson, James Shorter, Yong-Jie Zhang, Leonard Petrucelli
Language: English
Subfield: Clinical Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: green
Awards: Programming human protein disaggregases against FTD, Pathophysiology of genetically defined dementia and neurodegeneration: Defining therapeutic targets and pathways, Pathobiology of Neurodegeneration in C9ORF72 repeat expansion, Defining mechanisms of AAA+ disaggregases, Silencing C9or72 with rAAV Mediated RNAi, Expanding insights into FTD disease mechanisms
OpenAlex ID: W3082060010