A Druglike Small Molecule that Targets r(CCUG) Repeats in Myotonic Dystrophy Type 2 Facilitates Degradation by RNA Quality Control Pathways.

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作者： Sarah,Wagner-Griffin ^[1] ; Masahito,Abe ^[1] ; Raphael I,Benhamou ^[1] ; Alicia J,Angelbello ^[1] ; Kamalakannan,Vishnu ^[1] ; Jonathan L,Chen ^[1] ; Jessica L,Childs-Disney ^[1] ; Matthew D,Disney ^[1]

作者单位： Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States. ^[1]

主题词苯并噻唑类(Benzothiazoles);人类(Humans);分子结构(Molecular Structure);营养不良性肌强直(Myotonic Dystrophy);喹唑啉类(Quinazolines);RNA(RNA);RNA结合蛋白质类(RNA-Binding Proteins);重复序列, 核酸(Repetitive Sequences, Nucleic Acid);小分子化合物库(Small Molecule Libraries);构效关系(Structure-Activity Relationship)

DOI 10.1021/acs.jmedchem.1c00414

PMID 34101465

发布时间 2022-07-16

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Journal of medicinal chemistry

2021年64卷12期

8474-8485页

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A Druglike Small Molecule that Targets r(CCUG) Repeats in Myotonic Dystrophy Type 2 Facilitates Degradation by RNA Quality Control Pathways.

Journal of medicinal chemistry

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Journal of medicinal chemistry

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A Druglike Small Molecule that Targets r(CCUG) Repeats in Myotonic Dystrophy Type 2 Facilitates Degradation by RNA Quality Control Pathways.

Journal of medicinal chemistry

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Journal of medicinal chemistry

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