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

Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide

使用针对 CAG 重复序列的反义寡核苷酸抑制 SCA3 和 SCA1 小鼠中的突变蛋白表达

Eleni Kourkouta, Rudie Weij, Anchel González-Barriga, Melissa Mulder, Ruurd Verheul, Sieto Bosgra, Bas Groenendaal, Jukka Puoliväli, Jussi Toivanen, Judith C T van Deutekom, Nicole A Datson

Intracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro

体外肌母细胞和分化肌管细胞中反义寡核苷酸在非辅助摄取后的细胞内分布和核活性

González-Barriga, Anchel; Nillessen, Bram; Kranzen, Julia; van Kessel, Ingeborg D G; Croes, Huib J E; Aguilera, Begoña; de Visser, Peter C; Datson, Nicole A; Mulders, Susan A M; van Deutekom, Judith C T; Wieringa, Bé; Wansink, Derick G

The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain

亨廷顿基因中 CAG 重复扩增是整个亨廷顿氏病小鼠大脑中治疗性 RNA 调节的靶点

Nicole A Datson, Anchel González-Barriga, Eleni Kourkouta, Rudie Weij, Jeroen van de Giessen, Susan Mulders, Outi Kontkanen, Taneli Heikkinen, Kimmo Lehtimäki, Judith C T van Deutekom

Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy

数字液滴PCR技术用于绝对定量杜氏肌营养不良症(临床前)开发中反义寡核苷酸诱导的外显子跳跃

Verheul, Ruurd C; van Deutekom, Judith C T; Datson, Nicole A

Cell membrane integrity in myotonic dystrophy type 1: implications for therapy

型强直性肌营养不良症的细胞膜完整性:对治疗的意义

Anchel González-Barriga, Julia Kranzen, Huib J E Croes, Suzanne Bijl, Walther J A A van den Broek, Ingeborg D G van Kessel, Baziel G M van Engelen, Judith C T van Deutekom, Bé Wieringa, Susan A M Mulders, Derick G Wansink

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

2OMePS 反义寡核苷酸治疗 mdx 小鼠后化合物、转录本和蛋白质效应的动态

Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus

Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy

泼尼松龙治疗不会干扰杜氏肌营养不良症中 2'-O-甲基硫代磷酸酯反义介导的外显子跳跃

Ingrid E C Verhaart, Hans Heemskerk, Tatyana G Karnaoukh, Ingrid G M Kolfschoten, Anne Vroon, Gert-Jan B van Ommen, Judith C T van Deutekom, Annemieke Aartsma-Rus

Targeting several CAG expansion diseases by a single antisense oligonucleotide.

利用单个反义寡核苷酸靶向治疗多种 CAG 扩增疾病

Evers Melvin M, Pepers Barry A, van Deutekom Judith C T, Mulders Susan A M, den Dunnen Johan T, Aartsma-Rus Annemieke, van Ommen Gert-Jan B, van Roon-Mom Willeke M C

Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model

在 mdx 小鼠模型中对 2'-O-甲基硫代磷酸酯 RNA 反义寡核苷酸进行临床前药代动力学和药效学研究

Heemskerk, Hans; de Winter, Christa; van Kuik, Petra; Heuvelmans, Niki; Sabatelli, Patrizia; Rimessi, Paola; Braghetta, Paola; van Ommen, Gert-Jan B; de Kimpe, Sjef; Ferlini, Alessandra; Aartsma-Rus, Annemieke; van Deutekom, Judith C T

Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms

反义寡核苷酸设计指南及剪接调控机制解析

Aartsma-Rus, Annemieke; van Vliet, Laura; Hirschi, Marscha; Janson, Anneke A M; Heemskerk, Hans; de Winter, Christa L; de Kimpe, Sjef; van Deutekom, Judith C T; 't Hoen, Peter A C; van Ommen, Gert-Jan B