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

Vector-Free Deep Tissue Targeting of DNA/RNA Therapeutics via Single Capacitive Discharge Conductivity-Clamped Gene Electrotransfer.

通过单电容放电电导钳位基因电转移实现无载体深层组织靶向DNA/RNA疗法

Pinyon Jeremy L, von Jonquieres Georg, Mow Stephen L, Abed Amr Al, Lai Keng-Yin, Manoharan Mathumathi, Crawford Edward N, Xue Stanley H, Smith-Moore Sarah, Caproni Lisa J, Milsom Sarah, Klugmann Matthias, Lovell Nigel H, Housley Gary D

Engineering oxypurinol-responsive riboswitches based on bacterial xanthine aptamers for gene expression control in mammalian cell culture

基于细菌黄嘌呤适体的氧嘌呤醇响应核糖开关工程化用于哺乳动物细胞培养中的基因表达控制

Hedwig, Vera; Spöring, Maike; Ottlinger, Julia; Köse, Sila; Nar, Herbert; Schnapp, Gisela; Gottschling, Dirk; Klein, Holger; Aspnes, Gary; Klugmann, Matthias; Hartig, Jörg S

Preclinical development of TAK-754, a high-performance AAV8-based vector expressing coagulation factor VIII

TAK-754 的临床前开发,TAK-754 是一种基于 AAV8 的高性能载体,可表达凝血因子 VIII

Lengler, Johannes; Weiller, Markus; Horling, Franziska; Mayrhofer, Josef; Schuster, Maria; Falkner, Falko G; Gil-Farina, Irene; Klugmann, Matthias; Scheiflinger, Friedrich; Hoellriegl, Werner; Rottensteiner, Hanspeter

Gene Electrotransfer via Conductivity-Clamped Electric Field Focusing Pivots Sensori-Motor DNA Therapeutics: "A Spoonful of Sugar Helps the Medicine Go Down"

利用电导钳制电场聚焦进行基因电转移,为感觉运动DNA疗法带来转机:“一勺糖能让药更容易下咽”

Pinyon, Jeremy L; von Jonquieres, Georg; Crawford, Edward N; Abed, Amr Al; Power, John M; Klugmann, Matthias; Browne, Cherylea J; Housley, David M; Wise, Andrew K; Fallon, James B; Shepherd, Robert K; Lin, John Y; McMahon, Catherine; McAlpine, David; Birman, Catherine S; Lai, Waikong; Enke, Ya Lang; Carter, Paul M; Patrick, James F; Gay, Robert D; Marie, Corinne; Scherman, Daniel; Lovell, Nigel H; Housley, Gary D

TRPC Channels Activated by G Protein-Coupled Receptors Drive Ca(2+) Dysregulation Leading to Secondary Brain Injury in the Mouse Model

G蛋白偶联受体激活的TRPC通道驱动Ca(2+)失调,导致小鼠模型继发性脑损伤

Parmar, Jasneet; von Jonquieres, Georg; Gorlamandala, Nagarajesh; Chung, Brandon; Craig, Amanda J; Pinyon, Jeremy L; Birnbaumer, Lutz; Klugmann, Matthias; Moorhouse, Andrew J; Power, John M; Housley, Gary D

Recessive aminoacyl-tRNA synthetase disorders: lessons learned from in vivo disease models

隐性氨酰tRNA合成酶疾病:从体内疾病模型中汲取的经验教训

Kalotay, Elizabeth; Klugmann, Matthias; Housley, Gary D; Fröhlich, Dominik

Editorial: Myelin Repair: At the Crossing-Lines of Myelin Biology and Gene Therapy

社论:髓鞘修复:髓鞘生物学与基因治疗的交叉领域

Fröhlich, Dominik; Gessler, Dominic J; Klugmann, Matthias

Correction to: Developmental delay and late onset HBSL pathology in hypomorphic Dars1(M256L) mice

更正:Dars1(M256L)低表达小鼠的发育迟缓和迟发性HBSL病理

Klugmann, Matthias; Kalotay, Elizabeth; Delerue, Fabien; Ittner, Lars M; Bongers, Andre; Yu, Josephine; Morris, Margaret J; Housley, Gary D; Fröhlich, Dominik

Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

最小必需人凝血因子VIII改变可增强分泌和基因治疗效率

Cao, Wenjing; Dong, Biao; Horling, Franziska; Firrman, Jenni A; Lengler, Johannes; Klugmann, Matthias; de la Rosa, Maurus; Wu, Wenman; Wang, Qizhao; Wei, Hongying; Moore, Andrea R; Roberts, Sean A; Booth, Carmen J; Hoellriegl, Werner; Li, Dong; Konkle, Barbara; Miao, Carol; Reipert, Birgit M; Scheiflinger, Friedrich; Rottensteiner, Hanspeter; Xiao, Weidong

The Leukodystrophies HBSL and LBSL-Correlates and Distinctions

白质营养不良症 HBSL 和 LBSL——相关性和区别

Muthiah, Annapoorani; Housley, Gary D; Klugmann, Matthias; Fröhlich, Dominik