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 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

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

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

rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy

rAAV 相关疗法完全挽救了 X 连锁肌管性肌病中的肌核和肌丝功能

Jacob A Ross, Hichem Tasfaout, Yotam Levy, Jennifer Morgan, Belinda S Cowling, Jocelyn Laporte, Edmar Zanoteli, Norma B Romero, Dawn A Lowe, Heinz Jungbluth, Michael W Lawlor, David L Mack, Julien Ochala

Differential physiological roles for BIN1 isoforms in skeletal muscle development, function and regeneration

BIN1 亚型在骨骼肌发育、功能和再生中的不同生理作用

Ivana Prokic, Belinda S Cowling, Candice Kutchukian, Christine Kretz, Hichem Tasfaout, Vincent Gache, Josiane Hergueux, Olivia Wendling, Arnaud Ferry, Anne Toussaint, Christos Gavriilidis, Vasugi Nattarayan, Catherine Koch, Jeanne Lainé, Roy Combe, Laurent Tiret, Vincent Jacquemond, Fanny Pilot-Stor

Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice

单次肌内注射AAV-shRNA可降低小鼠DNM2表达并预防肌管性肌病

Tasfaout, Hichem; Lionello, Valentina M; Kretz, Christine; Koebel, Pascale; Messaddeq, Nadia; Bitz, Deborah; Laporte, Jocelyn; Cowling, Belinda S

Reducing dynamin 2 (DNM2) rescues DNM2-related dominant centronuclear myopathy

降低动力蛋白2 (DNM2) 水平可挽救DNM2相关显性中心核肌病

Buono, Suzie; Ross, Jacob A; Tasfaout, Hichem; Levy, Yotam; Kretz, Christine; Tayefeh, Leighla; Matson, John; Guo, Shuling; Kessler, Pascal; Monia, Brett P; Bitoun, Marc; Ochala, Julien; Laporte, Jocelyn; Cowling, Belinda S

Centronuclear myopathies under attack: A plethora of therapeutic targets

中心核肌病面临诸多挑战:治疗靶点众多

Tasfaout, Hichem; Cowling, Belinda S; Laporte, Jocelyn