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

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

反义寡核苷酸介导的肌肉糖原合成酶1合成抑制作为庞贝病底物减少疗法的一种方法

Clayton, Nicholas P; Nelson, Carol A; Weeden, Timothy; Taylor, Kristin M; Moreland, Rodney J; Scheule, Ronald K; Phillips, Lucy; Leger, Andrew J; Cheng, Seng H; Wentworth, Bruce M

Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease

庞贝病小鼠模型中糖原代谢多个方面的失调

Kristin M Taylor, Elizabeth Meyers, Michael Phipps, Priya S Kishnani, Seng H Cheng, Ronald K Scheule, Rodney J Moreland

Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease

在2型戈谢病小鼠模型中,全身性给予葡糖基神经酰胺合成酶抑制剂可减少中枢神经系统底物并延长寿命。

Cabrera-Salazar, Mario A; Deriso, Matthew; Bercury, Scott D; Li, Lingyun; Lydon, John T; Weber, William; Pande, Nilesh; Cromwell, Mandy A; Copeland, Diane; Leonard, John; Cheng, Seng H; Scheule, Ronald K

Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression

尽管肝脏表达水平较低,但全身性注射AAV8-α-半乳糖苷酶A仍可在非人灵长类动物中诱导体液耐受。

Nietupski, Jennifer B; Hurlbut, Gregory D; Ziegler, Robin J; Chu, Qiuming; Hodges, Bradley L; Ashe, Karen M; Bree, Mark; Cheng, Seng H; Gregory, Richard J; Marshall, John; Scheule, Ronald K

Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease

基于亚氨基糖的葡糖基神经酰胺合成酶抑制剂可增加桑德霍夫病小鼠模型的脑糖鞘脂含量并提高其存活率。

Ashe, Karen M; Bangari, Dinesh; Li, Lingyun; Cabrera-Salazar, Mario A; Bercury, Scott D; Nietupski, Jennifer B; Cooper, Christopher G F; Aerts, Johannes M F G; Lee, Edward R; Copeland, Diane P; Cheng, Seng H; Scheule, Ronald K; Marshall, John

Surfactant protein-d inhibits lung inflammation caused by ventilation in premature newborn lambs

表面活性蛋白-d抑制早产新生羔羊因通气引起的肺部炎症

Sato, Atsuyasu; Whitsett, Jeffrey A; Scheule, Ronald K; Ikegami, Machiko

The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways

使用羧甲基纤维素凝胶增加小鼠气道中的非病毒基因转移

Uta Griesenbach, Cuixiang Meng, Raymond Farley, Marguerite Y Wasowicz, Felix M Munkonge, Mario Chan, Charlotte Stoneham, Stephanie G Sumner-Jones, Ian A Pringle, Deborah R Gill, Stephen C Hyde, Barbara Stevenson, Emma Holder, Hiroshi Ban, Mamoru Hasegawa, Seng H Cheng, Ronald K Scheule, Patrick L Si

Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice

评估全身性卵泡抑素作为佐剂刺激庞贝病小鼠肌肉修复和改善运动功能的效果

Joseph W Foley, Scott D Bercury, Patrick Finn, Seng H Cheng, Ronald K Scheule, Robin J Ziegler

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy

预先存在的免疫力和灵长类动物体内低表达水平凸显了肝脏靶向AAV8介导的基因治疗在转化应用方面面临的挑战。

Hurlbut, Gregory D; Ziegler, Robin J; Nietupski, Jennifer B; Foley, Joseph W; Woodworth, Lisa A; Meyers, Elizabeth; Bercury, Scott D; Pande, Nilesh N; Souza, David W; Bree, Mark P; Lukason, Michael J; Marshall, John; Cheng, Seng H; Scheule, Ronald K

Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy

利用酶和底物减少疗法改善1型戈谢病小鼠模型中溶酶体葡糖基神经酰胺水平的管理

Marshall, John; McEachern, Kerry Anne; Chuang, Wei-Lien; Hutto, Elizabeth; Siegel, Craig S; Shayman, James A; Grabowski, Greg A; Scheule, Ronald K; Copeland, Diane P; Cheng, Seng H