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

Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA

针对神经退行性疾病的基因靶向疗法:ALS和SMA的策略和意义

Na, Yue; Bai, Jinku; Zhang, Ning; Geng, Fang; Wang, Xijun; González-Cidad, Aitor; García-Moncó, Juan Carlos; Román, Gustavo C; Yesbek Kaymaz, Ayse; Bora-Akoğlu, Gamze; Erdem Yurter, Hayat; Grunseich, Christopher

Locoregional Delivery of miRNAs for Glioblastoma Treatment: A Systematic Review of Advances in Delivery Systems

miRNA局部区域递送治疗胶质母细胞瘤:递送系统进展的系统性综述

Wieners, Luc; Garcia, Martin E; Yang, Xiaowen; Wu, Shiyue; Dou, Zhiya; Dong, Yuxiao; Dai, Jundong; Yesbek Kaymaz, Ayse; Bora-Akoğlu, Gamze; Erdem Yurter, Hayat; Grunseich, Christopher; Liu, Zhe; Wei, Xuetuan; Pan, Qingyan; Zuo, Xifeng; Chen, Ping; Guo, Ailing; Priya Dharshini, Loganathan Chandramani; Gaiamova, Elizaveta; Serreau, Raphael; Garcion, Emmanuel; Morisset-Lopez, Severine; Baril, Patrick

PROTACs therapeutically target the polyglutamine androgen receptor in spinal and bulbar muscular atrophy models.

PROTACs 以脊髓延髓肌萎缩症模型中的多聚谷氨酰胺雄激素受体为治疗靶点。

Sangotra Agamjot, Reddy Satya L, Kuo Curtis J, Xiang Weiguo, Merry Diane E, Grunseich Christopher, Wang Shaomeng, Lieberman Andrew P

CSF and blood neuronal injury biomarkers in spinal bulbar muscular atrophy and amyotrophic lateral sclerosis 4

脊髓延髓肌萎缩症和肌萎缩侧索硬化症4中脑脊液和血液神经元损伤生物标志物

Shahim, Pashtun; AlQahtani, Abdullah; Kokkinis, Angela D; Kazmi, Narjis; Ezuma-Ngwu, Marie; Misra, Jahan; Harmison, George; Benoit, Nicole; Jones, Melina; Howe, Elizabeth; Schindler, Alice B; Joe, Galen O; Grunseich, Christopher

NAD(+) dyshomeostasis in RYR1-related myopathies

RYR1相关肌病中的NAD(+)稳态失衡

Lawal, Tokunbor A; Riekhof, Willa; Groom, Linda; Varma, Pooja; Chrismer, Irene C; Kokkinis, Angela; Grunseich, Christopher; Witherspoon, Jessica W; Razaqyar, Muslima S; Sinaii, Ninet; Meilleur, Katherine G; Xiang, Lichen; Buzkova, Jana; Euro, Liliya; Mohassel, Payam; Dirksen, Robert T; Todd, Joshua J

Allele-specific silencing of a dominant SETX mutation in familial amyotrophic lateral sclerosis type 4

家族性肌萎缩侧索硬化症4型中显性SETX突变的等位基因特异性沉默

Winkelsas, Audrey; Apfel, Athena; Johnson, Brian; Harmison, George; Perez, Kimberly Diaz; Li, Dongjun; Cheung, Vivian G; Grunseich, Christopher

The enhancer RNA, AANCR, regulates APOE expression in astrocytes and microglia

增强子RNA AANCR调控星形胶质细胞和小胶质细胞中APOE的表达。

Wan, Ma; Liu, Yaojuan; Li, Dongjun; Snyder, Ryan J; Elkin, Lillian B; Day, Christopher R; Rodriguez, Joseph; Grunseich, Christopher; Mahley, Robert W; Watts, Jason A; Cheung, Vivian G

Rycal S48168 (ARM210) for RYR1-related myopathies: a phase one, open-label, dose-escalation trial

Rycal S48168 (ARM210) 用于治疗 RYR1 相关肌病:一项 I 期、开放标签、剂量递增试验

Todd, Joshua J; Lawal, Tokunbor A; Chrismer, Irene C; Kokkinis, Angela; Grunseich, Christopher; Jain, Minal S; Waite, Melissa R; Biancavilla, Victoria; Pocock, Shavonne; Brooks, Kia; Mendoza, Christopher J; Norato, Gina; Cheung, Ken; Riekhof, Willa; Varma, Pooja; Colina-Prisco, Claudia; Emile-Backer, Magalie; Meilleur, Katherine G; Marks, Andrew R; Webb, Yael; Marcantonio, Eugene E; Foley, A Reghan; Bönnemann, Carsten G; Mohassel, Payam

Functional Outcome Measures to Optimize Drug Development in Spinal and Bulbar Muscular Atrophy: Results From a Meta-Analysis of the Global SBMA Dataset

功能性结局指标在脊髓延髓肌萎缩症药物研发优化中的应用:全球 SBMA 数据集荟萃分析结果

Huggett, Spencer B; Tebbenkamp, Andrew T N; Rinaldi, Carlo; Jayaseelan, Dipa; Zampedri, Luca; Blasi, Lorenzo; Fortuna, Andrea; Alqahtani, Abdullah; Kokkinis, Angela; Dahlqvist, Julia; Fenu, Silvia; Cavalca, Eleonora; Bertini, Alessandro; Mariotti, Caterina; Grunseich, Christopher; Kawase, Takahiro; Kishimoto, Yoshiyuki; Yamada, Shinichiro; Katsuno, Masahisa; Fratta, Pietro; Conte, Amelia; Sabatelli, Mario; Soraru, Gianni; Vissing, John; Kang, Minsung; Park, Jin-Sung; Pareyson, Davide; Viglietta, Vissia

Protein biomarker signature in patients with spinal and bulbar muscular atrophy

脊髓延髓肌萎缩症患者的蛋白质生物标志物特征

Tebbenkamp, Andrew Tn; Huggett, Spencer B; Lombardi, Vittoria; Zampedri, Luca; AlQahtani, Abdullah; Kokkinis, Angela; Malaspina, Andrea; Rinaldi, Carlo; Grunseich, Christopher; Fratta, Pietro; Viglietta, Vissia