换一批
换一批Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon.
作者：
关键词
AONATXN3Ataxin-3CAG repeatDMDDuchenne muscular dystrophyExon skippingICVMJDMachado–Joseph diseaseNESNLSPolyQPolyglutamine disorderPolyglutamine repeatRNA interferenceRNAiSCA3SNPSpinocerebellar ataxia type 3UIMsVCPantisense oligonucleotideataxin-3intra-cerebral ventricularnuclear export signalnuclear localization signalpolyglutaminesingle nucleotide polymorphismspinocerebellar ataxia type 3ubiquitin interacting motifsvalosin containing protein
主题词
动物(Animals);细胞, 培养的(Cells, Cultured);DNA突变分析(DNA Mutational Analysis);剂量效应关系, 药物(Dose-Response Relationship, Drug);外显子(Exons);成纤维细胞(Fibroblasts);人类(Humans);Machado-Joseph病(Machado-Joseph Disease);小鼠(Mice);小鼠, 近交C57BL(Mice, Inbred C57BL);突变(Mutation);神经组织蛋白质类(Nerve Tissue Proteins);核蛋白质类(Nuclear Proteins);寡核苷酸类, 反义(Oligonucleotides, Antisense);肽类(Peptides);蛋白质结合(Protein Binding);RNA, 信使(RNA, Messenger);阻遏蛋白质类(Repressor Proteins);转染(Transfection);三核苷酸重复(Trinucleotide Repeats);泛素(Ubiquitin)
DOI
10.1016/j.nbd.2013.04.019
PMID
23659897
发布时间
2022-03-18
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Neurobiology of disease
49-56页
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