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

Author Correction: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

作者更正:基于腺相关病毒(AAV)的RNAi靶向ataxin-2可改善TDP-43小鼠的存活率和病理状况。

Amado, Defne A; Robbins, Ashley B; Whiteman, Katherine R; Smith, Alicia R; Chillon, Guillem; Chen, Yonghong; Fuller, Joshua A; Patty, Nicholas A; Izda, Aleksandar; Cheng, Congsheng; Nelson, Shareen; Dichter, Abigail I; Mazzoni, Esteban O; Monteys, Alex Mas; Davidson, Beverly L

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice.

基于 AAV 的 RNAi 递送靶向 ataxin-2 可改善 TDP-43 小鼠的生存率和病理状况

Amado Defne A, Robbins Ashley B, Whiteman Katherine R, Smith Alicia R, Chillon Guillem, Chen Yonghong, Fuller Joshua A, Patty Nicholas A, Izda Aleksandar, Cheng Congsheng, Nelson Shareen, Dichter Abigail I, Mazzoni Esteban O, Monteys Alex Mas, Davidson Beverly L

AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALS

基于腺相关病毒载体(AAV)的RNAi递送靶向Ataxin-2可改善快速进展型和缓慢进展型散发性肌萎缩侧索硬化症(ALS)小鼠模型的生存率、力量和病理状况。

Amado, Defne A; Robbins, Ashley B; Smith, Alicia R; Whiteman, Katherine R; Chillon Bosch, Guillem; Chen, Yonghong; Fuller, Joshua A; Izda, Aleksandar; Nelson, Shareen; Dichter, Abigail I; Monteys, Alex Mas; Davidson, Beverly L

Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing

利用长读长测序技术对扩增的亨廷顿等位基因进行单倍型SNP分析,以实现等位基因特异性基因编辑。

Fang, Li; Monteys, Alex Mas; Dürr, Alexandra; Keiser, Megan; Cheng, Congsheng; Harapanahalli, Akhil; Gonzalez-Alegre, Pedro; Davidson, Beverly L; Wang, Kai

Erratum: Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing

勘误:利用长读长测序技术对扩增的亨廷顿等位基因进行等位基因特异性基因编辑的单倍型SNP进行分析

Fang, Li; Monteys, Alex Mas; Dürr, Alexandra; Keiser, Megan; Cheng, Congsheng; Harapanahalli, Akhil; Gonzalez-Alegre, Pedro; Davidson, Beverly L; Wang, Kai

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

Limited astrocyte-to-neuron conversion in the mouse brain using NeuroD1 overexpression

利用NeuroD1过表达限制小鼠脑内星形胶质细胞向神经元的转化

Leib, David; Chen, Yong Hong; Monteys, Alex Mas; Davidson, Beverly L

DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencing

DeepRepeat:直接定量纳米孔测序信号数据中的短串联重复序列

Fang, Li; Liu, Qian; Monteys, Alex Mas; Gonzalez-Alegre, Pedro; Davidson, Beverly L; Wang, Kai

Regulated control of gene therapies by drug-induced splicing

通过药物诱导剪接调控基因治疗

Alex Mas Monteys, Amiel A Hundley, Paul T Ranum, Luis Tecedor, Amy Muehlmatt, Euyn Lim, Dmitriy Lukashev, Rajeev Sivasankaran, Beverly L Davidson

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

利用 CRISPR/Cas9 在体内和体外编辑突变的 Huntingtin 等位基因

Alex Mas Monteys, Shauna A Ebanks, Megan S Keiser, Beverly L Davidson