C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy

Authors: Casey 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 Srubek Tomassy, Alexander McCampbell, Frank Rigo, Dennis W. Dickson, James Shorter, Yong‐Jie Zhang, Leonard Petrucelli - Science Translational Medicine 2020 cited by 196

-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.