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

First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial

首次人体高剂量AAV9鞘内基因疗法治疗儿童CLN7疾病:一项1期、开放标签、单次递增剂量、非随机临床试验

Greenberg, Benjamin M; Minassian, Berge; Messahel, Souad; Edgar, Veronica Bordes; Lowden, Andrea; Dahshi, Hamza; Nettesheim, Emily R; Nguyen, Hoang H; Hughes, Samuel; Muthukumar, Alagar R; Srinivasan, Kalayarasan; Iannaccone, Susan; Varadarajan, Ganapathy; Gray, Steven J; Kayani, Saima N

Biomarker-Based Pharmacological Characterization of ENX-102, a Novel α2/3/5 Subtype-Selective GABAA Receptor Positive Allo-Steric Modulator: Translational Insights from Rodent and Human Studies

基于生物标志物的新型α2/3/5亚型选择性GABAA受体正向变构调节剂ENX-102的药理学表征:来自啮齿动物和人体研究的转化启示

Nettesheim, Pauline; Vadodaria, Krishna C; Vanover, Kimberly E; Borghans, Laura G J M; Arce, Estibaliz; Brubaker, William; Cunningham, Stephen; Parks, Stephanie; Serrats, Jordi; Sudarsan, Vikram; Taylor, Eve; Klaassen, Erica; Stuurman, Frederik E; Jacobs, Gabriel E

Enzymatic Modulation of the Pulmonary Glycocalyx Enhances Susceptibility to Streptococcus pneumoniae

肺糖萼的酶促调节增强了对肺炎链球菌的易感性

Goekeri, Cengiz; Linke, Kerstin A K; Hoffmann, Karen; Lopez-Rodriguez, Elena; Gluhovic, Vladimir; Voß, Anne; Kunder, Sandra; Zappe, Andreas; Timm, Sara; Nettesheim, Alina; Schickinger, Sebastian M K; Zobel, Christian M; Pagel, Kevin; Gruber, Achim D; Ochs, Matthias; Witzenrath, Martin; Nouailles, Geraldine

Neuronal ceroid lipofuscinoses type 7 (CLN7): a case series reporting cross sectional and retrospective clinical data to evaluate validity of standardized tools to assess disease progression, quality of life, and adaptive skills

神经元蜡样脂褐质沉积症7型(CLN7):一项病例系列研究,报告横断面和回顾性临床数据,以评估用于评估疾病进展、生活质量和适应能力的标准化工具的有效性

Kayani, Saima; BordesEdgar, Veronica; Lowden, Andrea; Nettesheim, Emily R; Dahshi, Hamza; Messahel, Souad; Minassian, Berge A; Greenberg, Benjamin M

Neuronal Ceroid Lipofuscinoses Type 7 (CLN7)- A Case Series Reporting Cross Sectional and Retrospective Clinical Data to Evaluate Validity of Standardized Tools to Assess Disease Progression, Quality of Life, and Adaptive Skills

神经元蜡样脂褐质沉积症 7 型 (CLN7) - 病例系列报告:横断面和回顾性临床数据,以评估用于评估疾病进展、生活质量和适应技能的标准化工具的有效性

Kayani, Saima; Edgar, Veronica Bordes; Lowden, Andrea; Nettesheim, Emily R; Dahshi, Hamza; Messahel, Souad; Minassian, Berge A; Greenberg, Benjamin M

Female sex hormones exacerbate retinal neurodegeneration

女性性激素会加剧视网膜神经退行性变

Rowe, Ashley A; Reyes, Sofia; Velasquez, Mauricio J; Yee, Tiffany; Nettesheim, Emily R; McDonald, Jeffrey G; Wert, Katherine J

Autophagy deficiency protects against ocular hypertension and neurodegeneration in experimental and spontanous glaucoma mouse models

自噬缺陷可预防实验性和自发性青光眼小鼠模型中的眼高压和神经退行性病变

Angela Dixon, Myoung Sup Shim, April Nettesheim, Aislyn Coyne, Chien-Chia Su, Haiyan Gong, Paloma B Liton

Effects of the G-quadruplex-binding drugs quarfloxin and CX-5461 on the malaria parasite Plasmodium falciparum

G-四链体结合药物 quarfloxin 和 CX-5461 对疟原虫恶性疟原虫的影响

Holly M Craven, Guilherme Nettesheim, Pietro Cicuta, Andrew M Blagborough, Catherine J Merrick

On the use of thermal forces to probe kinesin's response to force

利用热力探测驱动蛋白对力的响应

Chang, Chuan; Zheng, Tiantian; Nettesheim, Guilherme; Song, Hayoung; Cho, Changhyun; Crespi, Samuele; Shubeita, George

Shear stress induces autophagy in Schlemm's canal cells via primary cilia-mediated SMAD2/3 signaling pathway

剪切应力通过初级纤毛介导的 SMAD2/3 信号通路诱导 Schlemm 管细胞自噬

Myoung Sup Shim, Angela Dixon, April Nettesheim, Kristin M Perkumas, W Daniel Stamer, Yang Sun, Paloma B Liton