The Hairpin Form of r(G4C2)exp in c9ALS/FTD Is Repeat-Associated Non-ATG Translated and a Target for Bioactive Small Molecules.

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作者： Zi-Fu,Wang ^[1] ; Andrei,Ursu ^[1] ; Jessica L,Childs-Disney ^[1] ; Rea,Guertler ^[1] ; Wang-Yong,Yang ^[1] ; Viachaslau,Bernat ^[1] ; Suzanne G,Rzuczek ^[1] ; Rita,Fuerst ^[1] ; Yong-Jie,Zhang ^[2] ; Tania F,Gendron ^[2] ; Ilyas,Yildirim ^[3] ; Brendan G,Dwyer ^[1] ; Joseph E,Rice ^[4] ; Leonard,Petrucelli ^[2] ; Matthew D,Disney ^[5]

作者单位： Departments of Chemistry and Neuroscience, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. ^[1] Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA. ^[2] Department of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA. ^[3] Department of Medicinal Chemistry, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, 160 Frelinghuysen Road, Piscataway, NJ 08854, USA. ^[4] Departments of Chemistry and Neuroscience, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. Electronic address: disney@scripps.edu. ^[5]

关键词 RNA RNA folding amyotrophic lateral sclerosis c9ALS/FTD chemical biology drug design frontotemporal dementia nucleic acids small molecules

主题词肌萎缩侧索硬化(Amyotrophic Lateral Sclerosis);结合部位(Binding Sites);DNA重复序列扩增(DNA Repeat Expansion);额颞叶痴呆(Frontotemporal Dementia);人类(Humans);动力学(Kinetics);分子动力学模拟(Molecular Dynamics Simulation);核磁共振, 生物分子(Nuclear Magnetic Resonance, Biomolecular);核酸构象(Nucleic Acid Conformation);多核糖体(Polyribosomes);蛋白质生物合成(Protein Biosynthesis);RNA结合蛋白质类(RNA-Binding Proteins);小分子化合物库(Small Molecule Libraries);热力学(Thermodynamics)

DOI 10.1016/j.chembiol.2018.10.018

PMID 30503283

发布时间 2020-03-09

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Cell chemical biology

2019年26卷2期

179-190.e12页

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The Hairpin Form of r(G<sub>4</sub>C<sub>2</sub>)<sup>exp</sup> in c9ALS/FTD Is Repeat-Associated Non-ATG Translated and a Target for Bioactive Small Molecules.

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The Hairpin Form of r(G<sub>4</sub>C<sub>2</sub>)<sup>exp</sup> in c9ALS/FTD Is Repeat-Associated Non-ATG Translated and a Target for Bioactive Small Molecules.

Cell chemical biology

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Cell chemical biology

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