Huntington's disease: silencing a brutal killer.
作者信息
Pfister Edith L, Zamore Phillip D
机构信息
Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
出版信息
Exp Neurol. 2009 Dec;220(2):226-9. doi: 10.1016/j.expneurol.2009.09.017. Epub 2009 Sep 25.
Abstract
摘要
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本文引用的文献
1
Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.通过靶向mRNA中扩增的CAG重复序列实现突变型亨廷顿蛋白和ataxin-3基因的等位基因特异性沉默。
Nat Biotechnol. 2009 May;27(5):478-84. doi: 10.1038/nbt.1539. Epub 2009 May 3.
2
Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.针对三个单核苷酸多态性（SNP）的五种小干扰RNA（siRNA）可为四分之三的亨廷顿舞蹈症患者提供治疗。
Curr Biol. 2009 May 12;19(9):774-8. doi: 10.1016/j.cub.2009.03.030. Epub 2009 Apr 9.
3
Sustained effects of nonallele-specific Huntingtin silencing.非等位基因特异性亨廷顿蛋白沉默的持续效果。
Ann Neurol. 2009 Mar;65(3):276-85. doi: 10.1002/ana.21569.
4
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.大多数亨廷顿舞蹈症患者或许可用个体化的等位基因特异性RNA干扰进行治疗。
Exp Neurol. 2009 Jun;217(2):312-9. doi: 10.1016/j.expneurol.2009.03.004. Epub 2009 Mar 13.
5
CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup.亨廷顿舞蹈症基因中的CAG重复序列扩展与一个特定且可靶向的易感单倍群相关。
Am J Hum Genet. 2009 Mar;84(3):351-66. doi: 10.1016/j.ajhg.2009.02.003. Epub 2009 Feb 26.
6
MicroRNA-like off-target transcript regulation by siRNAs is species specific.小干扰RNA（siRNA）对类微小RNA脱靶转录本的调控具有物种特异性。
RNA. 2009 Feb;15(2):308-15. doi: 10.1261/rna.1326809.
7
Allele-specific silencing of mutant Huntington's disease gene.突变型亨廷顿舞蹈症基因的等位基因特异性沉默
J Neurochem. 2009 Jan;108(1):82-90. doi: 10.1111/j.1471-4159.2008.05734.x.
8
Linking SNPs to CAG repeat length in Huntington's disease patients.将亨廷顿舞蹈症患者的单核苷酸多态性与CAG重复序列长度关联起来。
Nat Methods. 2008 Nov;5(11):951-3. doi: 10.1038/nmeth.1261. Epub 2008 Oct 19.
9
Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease.突变型ataxin-3的等位基因特异性RNA沉默在Machado-Joseph病大鼠模型中介导神经保护作用。
PLoS One. 2008 Oct 8;3(10):e3341. doi: 10.1371/journal.pone.0003341.
10
Sequence- and target-independent angiogenesis suppression by siRNA via TLR3.小干扰RNA通过Toll样受体3实现不依赖序列和靶点的血管生成抑制
Nature. 2008 Apr 3;452(7187):591-7. doi: 10.1038/nature06765. Epub 2008 Mar 26.
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