Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALS. [electronic resource]
Producer: 20141103Description: 1043-50 p. digitalISSN:- 1097-4199
- 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
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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.
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