Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALS. [electronic resource]
- Neuron Sep 2014
- 1043-50 p. digital
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
1097-4199
10.1016/j.neuron.2014.07.041 doi
Adult Aged Amyotrophic Lateral Sclerosis--cerebrospinal fluid Animals Biomarkers--analysis C9orf72 Protein Cell Differentiation--drug effects Cells, Cultured Chlorocebus aethiops DNA Repeat Expansion--genetics Female Fibroblasts G-Quadruplexes Humans Male Middle Aged Nerve Tissue Proteins--metabolism Neurons--metabolism Protein Binding Proteins--chemistry RNA, Small Interfering--genetics