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

WITHDRAWN: Cell based dATP delivery as a therapy for chronic heart failure

撤回:基于细胞的dATP递送作为慢性心力衰竭的治疗方法

Mhatre, Ketaki N; Mathieu, Julie; Martinson, Amy; Flint, Galina; Blakley, Leslie P; Tabesh, Arash; Reinecke, Hans; Yang, Xiulan; Guan, Xuan; Murali, Eesha; Klaiman, Jordan M; Odom, Guy L; Brown, Mary Beth; Tian, Rong; Hauschka, Stephen D; Raftery, Daniel; Moussavi-Harami, Farid; Regnier, Michael; Murry, Charles E

Split intein-mediated protein trans-splicing to express large dystrophins

分裂内含肽介导的蛋白质反式剪接表达大型肌营养不良蛋白

Hichem Tasfaout, Christine L Halbert, Timothy S McMillen, James M Allen, Theodore R Reyes, Galina V Flint, Dirk Grimm, Stephen D Hauschka, Michael Regnier, Jeffrey S Chamberlain

Targeted CRISPR activation is functional in engineered human pluripotent stem cells but undergoes silencing after differentiation into cardiomyocytes and endothelium

靶向 CRISPR 激活在工程化人类多能干细胞中发挥作用,但在分化为心肌细胞和内皮细胞后会沉默

Elaheh Karbassi, Ruby Padgett, Alessandro Bertero, Hans Reinecke, Jordan M Klaiman, Xiulan Yang, Stephen D Hauschka, Charles E Murry1

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

Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model

老年临床前 DMD 动物模型中基因编辑和基因替换后肌营养不良蛋白表达的比较

Niclas E Bengtsson, Julie M Crudele, Jordan M Klaiman, Christine L Halbert, Stephen D Hauschka, Jeffrey S Chamberlain

Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.

肌营养不良蛋白基因编辑的稳定性取决于骨骼肌中的肌营养不良蛋白水平,但不取决于心肌中的肌营养不良蛋白水平

Bengtsson Niclas E, Tasfaout Hichem, Hauschka Stephen D, Chamberlain Jeffrey S

Laminin-111 Improves the Anabolic Response to Mechanical Load in Aged Skeletal Muscle

层粘连蛋白-111 改善老化骨骼肌对机械负荷的合成代谢反应

Koyal Garg, Ziad S Mahmassani, Svyatoslav Dvoretskiy, M Carmen Valero, Heather D Huntsman, Samuel Lapp, Yu-Fu Wu, Stephen D Hauschka, Dean J Burkin, Marni D Boppart

In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

在杜氏肌营养不良症小鼠模型中利用腺相关病毒载体对肌肉干细胞进行体内基因编辑

Jennifer B Kwon, Adarsh R Ettyreddy, Ashish Vankara, Joel D Bohning, Garth Devlin, Stephen D Hauschka, Aravind Asokan, Charles A Gersbach

Development of Novel Micro-dystrophins with Enhanced Functionality.

开发具有增强功能的新型微型肌营养不良蛋白

Ramos Julian N, Hollinger Katrin, Bengtsson Niclas E, Allen James M, Hauschka Stephen D, Chamberlain Jeffrey S

Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate.

基因疗法通过提高心肌细胞脱氧腺苷三磷酸水平挽救杜氏肌营养不良症小鼠的心脏功能障碍

Kolwicz Stephen C Jr, Hall John K, Moussavi-Harami Farid, Chen Xiolan, Hauschka Stephen D, Chamberlain Jeffrey S, Regnier Michael, Odom Guy L