Litcius/Paper detail

<i>C9orf72</i> poly(GR) aggregation induces TDP-43 proteinopathy

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

2020Science Translational Medicine197 citationsDOIOpen Access PDF

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.

Topics & Concepts

In vitroIn vivoCell biologyKey (lock)ChemistryBiophysicsBiologyBiochemistryGeneticsEcologyAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders ResearchHeterotopic Ossification and Related Conditions