Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration.
第一作者:
Clotilde,Lagier-Tourenne
第一单位:
Departments of Neurosciences, Cellular and Molecular Medicine, and Pathology, and Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093.
作者:
医学主题词
肌萎缩侧索硬化(Amyotrophic Lateral Sclerosis);动物(Animals);印迹法, DNA(Blotting, Southern);中枢神经系统(Central Nervous System);DNA引物(DNA Primers);DNA重复序列扩增(DNA Repeat Expansion);成纤维细胞(Fibroblasts);额颞叶变性(Frontotemporal Lobar Degeneration);基因型(Genotype);原位杂交, 荧光(In Situ Hybridization, Fluorescence);小鼠(Mice);寡核苷酸类, 反义(Oligonucleotides, Antisense);蛋白质类(Proteins);序列分析, RNA(Sequence Analysis, RNA)
DOI
10.1073/pnas.1318835110
PMID
24170860
发布时间
2024-01-04
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