• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利：CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验

相似文献

1
Oligonucleotide therapeutic approaches for Huntington disease.用于亨廷顿病的寡核苷酸治疗方法。
J Clin Invest. 2011 Feb;121(2):500-7. doi: 10.1172/JCI45130. Epub 2011 Feb 1.
2
Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.针对亨廷顿病基因中单核苷酸多态性的有效且选择性的反义寡核苷酸/突变亨廷顿蛋白的等位基因特异性沉默。
Mol Ther. 2011 Dec;19(12):2178-85. doi: 10.1038/mt.2011.201. Epub 2011 Oct 4.
3
Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.靶向扩展的 CAG 重复序列的反义寡核苷酸对突变亨廷顿表达的等位基因选择性抑制。
Biochemistry. 2010 Nov 30;49(47):10166-78. doi: 10.1021/bi101208k. Epub 2010 Nov 8.
4
An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases.寡核苷酸治疗策略在 polyQ 疾病中的评估。
BMC Mol Biol. 2012 Mar 7;13:6. doi: 10.1186/1471-2199-13-6.
5
Therapeutic Antisense Targeting of Huntingtin.亨廷顿病的治疗性反义靶向治疗。
DNA Cell Biol. 2020 Feb;39(2):154-158. doi: 10.1089/dna.2019.5188. Epub 2019 Dec 10.
6
Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification.通过反义寡核苷酸介导的蛋白质修饰来防止毒性 N 端亨廷顿片段的形成。
Nucleic Acid Ther. 2014 Feb;24(1):4-12. doi: 10.1089/nat.2013.0452. Epub 2013 Dec 31.
7
Systemic delivery of mutant huntingtin lowering antisense oligonucleotides to the brain using apolipoprotein A-I nanodisks for Huntington disease.利用载脂蛋白 A-I 纳米盘将突变亨廷顿病降低反义寡核苷酸递送到大脑系统。
J Control Release. 2024 Mar;367:27-44. doi: 10.1016/j.jconrel.2024.01.011. Epub 2024 Jan 24.
8
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.使用反义寡核苷酸对突变型亨廷顿蛋白进行等位基因特异性抑制：为所有亨廷顿病患者提供一种治疗选择。
PLoS One. 2014 Sep 10;9(9):e107434. doi: 10.1371/journal.pone.0107434. eCollection 2014.
9
Neurodegeneration. Huntington's research points to possible new therapies.神经退行性变。亨廷顿氏病的研究指向可能的新疗法。
Science. 2005 Oct 7;310(5745):43-5. doi: 10.1126/science.310.5745.43.
10
In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.候选等位基因特异性突变亨廷顿基因沉默反义寡核苷酸的体内评估
Mol Ther. 2014 Dec;22(12):2093-2106. doi: 10.1038/mt.2014.153. Epub 2014 Aug 7.

引用本文的文献

1
Molecular evolution of nucleoside deoxyribosyl transferase to enhance the activity toward 2'-fluoro-2'-deoxynucleoside.核苷脱氧核糖基转移酶的分子进化以增强对2'-氟-2'-脱氧核苷的活性。
J Ind Microbiol Biotechnol. 2024 Dec 31;52. doi: 10.1093/jimb/kuaf005.
2
Multifunctional Nanocarriers for Alzheimer's Disease: Befriending the Barriers.多功能纳米载体治疗阿尔茨海默病：突破障碍。
Mol Neurobiol. 2024 May;61(5):3042-3089. doi: 10.1007/s12035-023-03730-z. Epub 2023 Nov 15.
3
Neurodegenerative Diseases: New Hopes and Perspectives.神经退行性疾病：新的希望和展望。
Curr Mol Med. 2024;24(8):1004-1032. doi: 10.2174/1566524023666230907093451.
4
Nucleic acid drug vectors for diagnosis and treatment of brain diseases.用于脑疾病诊断和治疗的核酸药物载体。
Signal Transduct Target Ther. 2023 Jan 17;8(1):39. doi: 10.1038/s41392-022-01298-z.
5
A perspective on oligonucleotide therapy: Approaches to patient customization.寡核苷酸疗法的展望：患者个体化定制方法
Front Pharmacol. 2022 Oct 19;13:1006304. doi: 10.3389/fphar.2022.1006304. eCollection 2022.
6
Development of mAb-based polyglutamine-dependent and polyglutamine length-independent huntingtin quantification assays with cross-site validation.建立基于单抗的多聚谷氨酰胺依赖性和多聚谷氨酰胺长度非依赖性亨廷顿蛋白定量检测方法，并进行跨检测位点验证。
PLoS One. 2022 Apr 8;17(4):e0266812. doi: 10.1371/journal.pone.0266812. eCollection 2022.
7
AMD Genomics: Non-Coding RNAs as Biomarkers and Therapeutic Targets.年龄相关性黄斑变性基因组学：非编码RNA作为生物标志物和治疗靶点
J Clin Med. 2022 Mar 9;11(6):1484. doi: 10.3390/jcm11061484.
8
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.比较给药途径研究使用治疗性 siRNA 在 Dorset 绵羊中显示广泛的基因调节。
JCI Insight. 2021 Dec 22;6(24):e152203. doi: 10.1172/jci.insight.152203.
9
Therapeutic reversal of Huntington's disease by in vivo self-assembled siRNAs.体内自组装 siRNA 对亨廷顿病的治疗逆转。
Brain. 2021 Dec 16;144(11):3421-3435. doi: 10.1093/brain/awab354.
10
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.通过编辑编码区单核苷酸多态性杂合子实现突变亨廷顿蛋白的等位基因特异性敲低。
Hum Gene Ther. 2022 Jan;33(1-2):25-36. doi: 10.1089/hum.2020.323.

本文引用的文献

1
Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.通过转换为类似miRNA的RNAi机制实现亨廷顿蛋白表达的等位基因选择性抑制。
Chem Biol. 2010 Nov 24;17(11):1183-8. doi: 10.1016/j.chembiol.2010.10.013.
2
Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.靶向扩展的 CAG 重复序列的反义寡核苷酸对突变亨廷顿表达的等位基因选择性抑制。
Biochemistry. 2010 Nov 30;49(47):10166-78. doi: 10.1021/bi101208k. Epub 2010 Nov 8.
3
Alpha-synuclein suppression by targeted small interfering RNA in the primate substantia nigra.靶向小干扰 RNA 抑制灵长类动物黑质中的α-突触核蛋白。
PLoS One. 2010 Aug 11;5(8):e12122. doi: 10.1371/journal.pone.0012122.
4
Mammalian microRNAs predominantly act to decrease target mRNA levels.哺乳动物的 microRNAs 主要作用是降低靶 mRNA 水平。
Nature. 2010 Aug 12;466(7308):835-40. doi: 10.1038/nature09267.
5
Molecular mechanisms and potential therapeutical targets in Huntington's disease.亨廷顿病的分子机制及潜在治疗靶点。
Physiol Rev. 2010 Jul;90(3):905-81. doi: 10.1152/physrev.00041.2009.
6
RNAi-based treatment for neovascular age-related macular degeneration by Sirna-027.基于 RNAi 的 Sirna-027 治疗新生血管性年龄相关性黄斑变性。
Am J Ophthalmol. 2010 Jul;150(1):33-39.e2. doi: 10.1016/j.ajo.2010.02.006.
7
Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia.米泊美生，一种载脂蛋白 B 合成抑制剂，对家族性高胆固醇血症患者的低密度脂蛋白胆固醇的影响。
Am J Cardiol. 2010 May 15;105(10):1413-9. doi: 10.1016/j.amjcard.2010.01.003. Epub 2010 Mar 30.
8
A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus.一项针对呼吸道合胞病毒的基于 RNAi 的治疗的随机、双盲、安慰剂对照研究。
Proc Natl Acad Sci U S A. 2010 May 11;107(19):8800-5. doi: 10.1073/pnas.0912186107. Epub 2010 Apr 26.
9
Aberrant Rab11-dependent trafficking of the neuronal glutamate transporter EAAC1 causes oxidative stress and cell death in Huntington's disease.异常的 Rab11 依赖性神经元谷氨酸转运体 EAAC1 转运导致亨廷顿病中的氧化应激和细胞死亡。
J Neurosci. 2010 Mar 31;30(13):4552-61. doi: 10.1523/JNEUROSCI.5865-09.2010.
10
RNAi therapeutics for CNS disorders.中枢神经系统疾病的 RNAi 治疗。
Brain Res. 2010 Jun 18;1338:112-21. doi: 10.1016/j.brainres.2010.03.038. Epub 2010 Mar 20.

用于亨廷顿病的寡核苷酸治疗方法。

Oligonucleotide therapeutic approaches for Huntington disease.

机构信息

Alnylam Pharmaceuticals Inc., Cambridge, Massachusetts, USA.

出版信息

J Clin Invest. 2011 Feb;121(2):500-7. doi: 10.1172/JCI45130. Epub 2011 Feb 1.

DOI:10.1172/JCI45130
PMID:21285523
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3026739/
Abstract

Huntington disease is an autosomal dominant neurodegenerative disorder caused by a toxic expansion in the CAG repeat region of the huntingtin gene. Oligonucleotide approaches based on RNAi and antisense oligonucleotides provide promising new therapeutic strategies for direct intervention through reduced production of the causative mutant protein. Allele-specific and simultaneous mutant and wild-type allele-lowering strategies are being pursued with local delivery to the brain, each with relative merits. Delivery remains a key challenge for translational success, especially with chronic therapy. The potential of disease-modifying oligonucleotide approaches for Huntington disease will be revealed as they progress into clinical trials.

摘要

亨廷顿病是一种常染色体显性神经退行性疾病，由亨廷顿基因 CAG 重复区域的毒性扩张引起。基于 RNAi 和反义寡核苷酸的寡核苷酸方法为通过减少致病突变蛋白的产生提供了有前途的新治疗策略。正在通过局部递送到大脑来追求针对特定等位基因和同时降低突变型和野生型等位基因的策略，每种策略都有其相对的优点。对于转化成功来说，传递仍然是一个关键挑战，尤其是在慢性治疗中。随着疾病修饰寡核苷酸方法进入临床试验，它们在亨廷顿病中的潜力将逐渐显现。