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

Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease

将 CAG 碱基编辑策略转化为 CAA 可减少亨廷顿病中的致病突变。

Doo Eun Choi ,Jun Wan Shin ,Sophia Zeng ,Eun Pyo Hong ,Jae-Hyun Jang ,Jacob M Loupe ,Vanessa C Wheeler ,Hannah E Stutzman ,Ben Kleinstiver ,Jong-Min Lee

A method for the analysis of the oligomerization profile of the Huntington's disease-associated, aggregation-prone mutant huntingtin protein by isopycnic ultracentrifugation

一种通过等密度超速离心分析亨廷顿病相关、易聚集的突变亨廷顿蛋白寡聚化特征的方法

Raffaella Bonavita #, Rosaria Di Martino #, Giuseppe Cortone, Antonello Prodomo, Mariagrazia Di Gennaro, Gianluca Scerra, Valentino Panico, Silvia Nuzzo, Marco Salvatore, Sarah V Williams, Fulvia Vitale, Maria Gabriella Caporaso, Massimo D'Agostino, Francesca M Pisani, Angeleen Fleming, Maurizio Ren

Early detection of exon 1 huntingtin aggregation in zQ175 brains by molecular and histological approaches

通过分子和组织学方法早期检测 zQ175 脑中外显子 1 亨廷顿蛋白的聚集

Edward J Smith, Kirupa Sathasivam, Christian Landles, Georgina F Osborne, Michael A Mason, Casandra Gomez-Paredes, Bridget A Taxy, Rebecca E Milton, Anne Ast, Franziska Schindler, Chuangchuang Zhang, Wenzhen Duan, Erich E Wanker, Gillian P Bates

Semi-automated workflows to quantify AAV transduction in various brain areas and predict gene editing outcome for neurological disorders

半自动化工作流程可量化大脑各个区域的 AAV 转导并预测神经系统疾病的基因编辑结果

Fábio Duarte, Mergim Ramosaj, Ed Hasanovic, Sara Regio, Melanie Sipion, Maria Rey, Nicole Déglon

An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion

一种口服、可渗透大脑的小分子通过增强伪外显子的插入来降低亨廷顿蛋白的水平

Caroline Gubser Keller #, Youngah Shin #, Alex Mas Monteys #, Nicole Renaud, Martin Beibel, Natalia Teider, Thomas Peters, Thomas Faller, Sophie St-Cyr, Judith Knehr, Guglielmo Roma, Alejandro Reyes, Marc Hild, Dmitriy Lukashev, Diethilde Theil, Natalie Dales, Jang-Ho Cha, Beth Borowsky, Ricardo Dol

Alternative processing of human HTT mRNA with implications for Huntington's disease therapeutics

人类 HTT mRNA 的替代加工对亨廷顿氏病治疗具有重要意义

Sandra Fienko, Christian Landles, Kirupa Sathasivam, Sean J McAteer, Rebecca E Milton, Georgina F Osborne, Edward J Smith, Samuel T Jones, Marie K Bondulich, Emily C E Danby, Jemima Phillips, Bridget A Taxy, Holly B Kordasiewicz, Gillian P Bates

Differential Cellular Balance of Olfactory and Vomeronasal Epithelia in a Transgenic BACHD Rat Model of Huntington's Disease

亨廷顿氏病转基因 BACHD 大鼠模型中嗅觉和犁鼻上皮细胞的差异细胞平衡

Lina-Marielle Krysewski, Nicole Power Guerra, Annika Glatzel, Carsten Holzmann, Veronica Antipova, Oliver Schmitt, Libo Yu-Taeger, Huu Phuc Nguyen, Andreas Wree, Martin Witt

Global Rhes knockout in the Q175 Huntington's disease mouse model

Q175 亨廷顿氏病小鼠模型中的 Rhes 整体敲除

Taneli Heikkinen, Timo Bragge, Juha Kuosmanen, Teija Parkkari, Sanna Gustafsson, Mei Kwan, Jose Beltran, Afshin Ghavami, Srinivasa Subramaniam, Neelam Shahani, Uri Nimrod Ramírez-Jarquín, Larry Park, Ignacio Muñoz-Sanjuán, Deanna M Marchionini

Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

在非人类灵长类动物和大鼠中,纹状体内注射 AAV5-miHTT 具有良好的耐受性,并导致亨廷顿病病理关键区域出现 miHTT 转基因表达

Elisabeth A Spronck, Astrid Vallès, Margit H Lampen, Paula S Montenegro-Miranda, Sonay Keskin, Liesbeth Heijink, Melvin M Evers, Harald Petry, Sander J van Deventer, Pavlina Konstantinova, Martin de Haan

Genetic Screen in Adult Drosophila Reveals That dCBP Depletion in Glial Cells Mitigates Huntington Disease Pathology through a Foxo-Dependent Pathway

成年果蝇的基因筛查显示,神经胶质细胞中的 dCBP 耗竭可通过 Foxo 依赖性途径减轻亨廷顿病病理

Elodie Martin, Raheleh Heidari, Véronique Monnier, Hervé Tricoire