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

Proteomics-based evaluation of AAV dystrophin gene therapy outcomes in mdx skeletal muscle

基于蛋白质组学的AAV肌营养不良蛋白基因治疗在mdx骨骼肌中的疗效评估

Johnson, Erynn E; Reyes, Theodore R; Chamberlain, Jeffrey S; Ervasti, James M; Tasfaout, Hichem

Expression of full-length dystrophin reverses muscular dystrophy defects in young and old mdx4cv mice.

全长肌营养不良蛋白的表达可以逆转年轻和年老的 mdx4cv 小鼠的肌肉营养不良缺陷

Tasfaout Hichem, McMillen Timothy S, Reyes Theodore R, Halbert Christine L, Tian Rong, Regnier Michael, Chamberlain Jeffrey S

The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve

基因疗法的未来:更安全的载体,更精准的靶点:备受瞩目的失败案例需要进行深入的根本原因分析——但如果该领域愿意学习和发展,AAV的变革潜力仍然触手可及。

Chamberlain, Jeffrey S; Davidson, Beverly L; George, Lindsey A; Byrne, Barry J; Barrett, David

The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy

迈向AAV介导的杜氏肌营养不良症基因治疗之路

Bengtsson, Niclas E; Tasfaout, Hichem; Chamberlain, Jeffrey S

Correction: Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review

更正:基于肌营养不良蛋白和肌营养不良蛋白的杜氏肌营养不良症治疗方法:一项比较综述

Szwec, Sylwia; Kapłucha, Zuzanna; Chamberlain, Jeffrey S; Konieczny, Patryk

Genetic strategies for therapy of Duchenne muscular dystrophy

杜氏肌营养不良症的基因治疗策略

Łoboda, Agnieszka; Chamberlain, Jeffrey S; Dulak, Józef

Bidirectional role of Costameres in the pathophysiology of mdx skeletal muscles

肌节在 mdx 骨骼肌病理生理学中的双向作用

Banks, Glen B; Bisset, Darren R; Chamberlain, Jeffrey S

Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review

基于肌营养不良蛋白和肌营养不良蛋白受体的治疗方法在杜氏肌营养不良症治疗中的比较综述

Szwec, Sylwia; Kapłucha, Zuzanna; Chamberlain, Jeffrey S; Konieczny, Patryk

PCYT2-regulated lipid biosynthesis is critical to muscle health and ageing

PCYT2调控的脂质生物合成对肌肉健康和衰老至关重要。

Cikes, Domagoj; Elsayad, Kareem; Sezgin, Erdinc; Koitai, Erika; Torma, Ferenc; Orthofer, Michael; Yarwood, Rebecca; Heinz, Leonhard X; Sedlyarov, Vitaly; Miranda, Nasser Darwish; Taylor, Adrian; Grapentine, Sophie; Al-Murshedi, Fathiya; Abot, Anne; Weidinger, Adelheid; Kutchukian, Candice; Sanchez, Colline; Cronin, Shane J F; Novatchkova, Maria; Kavirayani, Anoop; Schuetz, Thomas; Haubner, Bernhard; Haas, Lisa; Hagelkruys, Astrid; Jackowski, Suzanne; Kozlov, Andrey V; Jacquemond, Vincent; Knauf, Claude; Superti-Furga, Giulio; Rullman, Eric; Gustafsson, Thomas; McDermot, John; Lowe, Martin; Radak, Zsolt; Chamberlain, Jeffrey S; Bakovic, Marica; Banka, Siddharth; Penninger, Josef M

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

在杜氏肌营养不良症GRMD模型中评估全身性AAV-微型肌营养不良蛋白基因治疗

Birch, Sharla M; Lawlor, Michael W; Conlon, Thomas J; Guo, Lee-Jae; Crudele, Julie M; Hawkins, Eleanor C; Nghiem, Peter P; Ahn, Mihye; Meng, Hui; Beatka, Margaret J; Fickau, Brittany A; Prieto, Juan C; Styner, Martin A; Struharik, Michael J; Shanks, Courtney; Brown, Kristy J; Golebiowski, Diane; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Clement, Nathalie; Coleman, Kirsten E; Corti, Manuela; Pan, Xiufang; Hauschka, Stephen D; Gonzalez, J Patrick; Morris, Carl A; Schneider, Joel S; Duan, Dongsheng; Chamberlain, Jeffrey S; Byrne, Barry J; Kornegay, Joe N