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

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

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。

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

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

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

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

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

Chronic pharmacologic manipulation of dopamine transmission ameliorates metabolic disturbance in Trappc9-linked brain developmental syndrome

长期药物控制多巴胺传递可改善 Trappc9 相关脑发育综合征的代谢紊乱

Yan Li, Muhammad Usman, Ellen Sapp, Yuting Ke, Zejian Wang, Adel Boudi, Marian DiFiglia, Xueyi Li

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

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

Nacc1 Mutation in Mice Models Rare Neurodevelopmental Disorder with Underlying Synaptic Dysfunction

小鼠中的 Nacc1 突变模型导致罕见的神经发育障碍,并伴有潜在的突触功能障碍

Mark A Deehan, Josine M Kothuis, Ellen Sapp, Kathryn Chase, Yuting Ke, Connor Seeley, Maria Iuliano, Emily Kim, Lori Kennington, Rachael Miller, Adel Boudi, Kai Shing, Xueyi Li, Edith Pfister, Christelle Anaclet, Michael Brodsky, Kimberly Kegel-Gleason, Neil Aronin, Marian DiFiglia

The Daily Experiences of Hispanic and Latinx Dementia Caregivers Study: Protocol for a Fully Remote Daily Diary Observational Cohort Study

西班牙裔和拉丁裔痴呆症照护者日常经历研究:一项完全远程每日日记观察队列研究方案

Mildrum Chana, Sofía; Álvarez, Lorelí; Poe, Abigail; Bibriescas, Natashia; Wang, Danny Hai; DiFiglia, Stephanie; Azuero, Andrés; Crowe, Michael; Puga, Frank

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

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

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

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