日期 影响因子
日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:
–

Mutant huntingtin exon 1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MutS Homolog 3 silencing and aggregation.

利用新表位抗体在小鼠脑中检测到突变亨廷顿蛋白外显子 1 蛋白：CAG 重复扩增、MutS 同源物 3 沉默和聚集的影响

期刊:Brain Communications
影响因子:4.5
doi:10.1093/braincomms/fcaf314
Sapp Ellen, Boudi Adel, Iwanowicz Andrew, Belgrad Jillian, Miller Rachael, Robertson Riannon, O'Reilly Daniel, Yamada Ken, Deng Yunping, Joni Marion, Li Xueyi, Kegel-Gleason Kimberly, Khvorova Anastasia, Reiner Anton, Aronin Neil, DiFiglia Marian
其它
Mouse
发表日期：2025
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Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington's disease pathogenesis in mice

通过RNA干扰抑制体细胞重复序列扩增和降低亨廷顿蛋白水平可协同预防小鼠亨廷顿病的发病机制。

期刊:
影响因子:
doi:10.1101/2025.06.24.661398
Belgrad, Jillian; Summers, Ashley; Landles, Christian; Greene, Jonathan R; Hildebrand, Samuel; Knox, Emily; Sapp, Ellen; Yamada, Nozomi; Furgal, Raymond; Miller, Rachael; Osborne, Georgina F; Chase, Kathryn; Luu, Eric; Freedman, Jason; Bramato, Brianna; McHugh, Nicholas; Benoit, Vicky; O'Reilly, Daniel; Greer, Paul; Bates, Gillian P; Vogt, Thomas F; Lee, Ramee; Howland, David; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia
细胞生物学
发表日期：2025
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Mutant Huntingtin exon1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MSH3 silencing, and aggregation

利用新表位抗体在小鼠脑中检测到突变型亨廷顿蛋白外显子1：CAG重复序列扩增、MSH3沉默和聚集的影响

期刊:
影响因子:
doi:10.1101/2024.12.31.630891
Sapp, Ellen; Boudi, Adel; Iwanowicz, Andrew; Belgrad, Jillian; Miller, Rachael; Robertson, Riannon; O'Reilly, Daniel; Yamada, Ken; Deng, Yunping; Joni, Marion; Li, Xueyi; Kegel-Gleason, Kimberly; Khvorova, Anastasia; Reiner, Anton; Aronin, Neil; DiFiglia, Marian
神经科学
多发性硬化症
发表日期：2025
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A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system.

可编程双靶向siRNA支架支持中枢神经系统中有效的双基因调控

期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkae368
Belgrad Jillian, Tang Qi, Hildebrand Sam, Summers Ashley, Sapp Ellen, Echeverria Dimas, O'Reilly Dan, Luu Eric, Bramato Brianna, Allen Sarah, Cooper David, Alterman Julia, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia
神经科学
发表日期：2024
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mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo.

mRNA 核聚集导致 siRNA 在体内对突变亨廷顿 mRNA 和蛋白质的沉默存在差异

期刊:Nucleic Acid Therapeutics
影响因子:4.7
doi:10.1089/nat.2024.0027
Allen Sarah, O'Reilly Daniel, Miller Rachael, Sapp Ellen, Summers Ashley, Paquette Joseph, Echeverria Moreno Dimas, Bramato Brianna, McHugh Nicholas, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia
免疫/内分泌
发表日期：2024
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Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca(2+) and Mg(2+) in the formulation

在制剂中添加Ca(2+)和Mg(2+)可预防中枢神经系统治疗性寡核苷酸的急性神经毒性

期刊:
影响因子:
doi:10.1101/2024.06.06.597639
Miller, Rachael; Paquette, Joseph; Barker, Alexandra; Sapp, Ellen; McHugh, Nicholas; Bramato, Brianna; Yamada, Nozomi; Alterman, Julia; Echeveria, Dimas; Yamada, Ken; Watts, Jonathan; Anaclet, Christelle; DiFiglia, Marian; Khvorova, Anastasia; Aronin, Neil
毒理研究
神经科学
发表日期：2024
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mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo.

mRNA 核聚集导致突变亨廷顿 mRNA 和蛋白质在体内通过 siRNA 沉默存在差异

期刊:
影响因子:
doi:10.1101/2024.04.24.590997
Allen Sarah, O'Reilly Daniel, Miller Rachael, Sapp Ellen, Summers Ashley, Paquette Joseph, Moreno Dimas Echeverria, Bramato Brianna, McHugh Nicholas, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia
免疫/内分泌
发表日期：2024
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Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease

二价siRNA介导的MSH3沉默可阻断亨廷顿病小鼠模型中的体细胞重复序列扩增

期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2023.09.016
O'Reilly, Daniel; Belgrad, Jillian; Ferguson, Chantal; Summers, Ashley; Sapp, Ellen; McHugh, Cassandra; Mathews, Ella; Boudi, Adel; Buchwald, Julianna; Ly, Socheata; Moreno, Dimas; Furgal, Raymond; Luu, Eric; Kennedy, Zachary; Hariharan, Vignesh; Monopoli, Kathryn; Yang, X William; Carroll, Jeffery; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia
细胞生物学
多发性硬化症
发表日期：2023
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A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system.

可编程双靶向二价siRNA支架支持中枢神经系统中有效的多基因调控

期刊:
影响因子:
doi:10.1101/2023.12.19.572404
Belgrad Jillian, Tang Qi, Hildebrand Sam, Summers Ashley, Sapp Ellen, Echeverria Dimas, O'Reilly Dan, Luu Eric, Bramato Brianna, Allen Sarah, Cooper David, Alterman Julia, Yamada Ken, Aronin Neil, DiFiglia Marian, Khvorova Anastasia
神经科学
发表日期：2023
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Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities

通过编码区单核苷酸多态性杂合性编辑实现突变亨廷顿蛋白的等位基因特异性敲低

期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2020.323
Oikemus, Sarah R; Pfister, Edith L; Sapp, Ellen; Chase, Kathryn O; Kennington, Lori A; Hudgens, Edward; Miller, Rachael; Zhu, Lihua Julie; Chaudhary, Akanksh; Mick, Eric O; Sena-Esteves, Miguel; Wolfe, Scot A; DiFiglia, Marian; Aronin, Neil; Brodsky, Michael H
发表日期：2022
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