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

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

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

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice

反义寡核苷酸介导的Dnm2基因敲低可预防和逆转小鼠肌管性肌病。

Tasfaout, Hichem; Buono, Suzie; Guo, Shuling; Kretz, Christine; Messaddeq, Nadia; Booten, Sheri; Greenlee, Sarah; Monia, Brett P; Cowling, Belinda S; Laporte, Jocelyn

Recessive mutations in the kinase ZAK cause a congenital myopathy with fibre type disproportion

ZAK激酶的隐性突变会导致先天性肌病,伴有肌纤维类型比例失调。

Vasli, Nasim; Harris, Elizabeth; Karamchandani, Jason; Bareke, Eric; Majewski, Jacek; Romero, Norma B; Stojkovic, Tanya; Barresi, Rita; Tasfaout, Hichem; Charlton, Richard; Malfatti, Edoardo; Bohm, Johann; Marini-Bettolo, Chiara; Choquet, Karine; Dicaire, Marie-Josée; Shao, Yi-Hong; Topf, Ana; O'Ferrall, Erin; Eymard, Bruno; Straub, Volker; Blanco, Gonzalo; Lochmüller, Hanns; Brais, Bernard; Laporte, Jocelyn; Tétreault, Martine