Department of Biochemistry, College of Medicine, Ewha Womans University, 1071 Anyangcheon-Ro, Yangcheon-Gu, Seoul, 07985, Republic of Korea.
Department of Health Sciences and Technology, Sungkyunkwan University, Seoul, Republic of Korea.
Muscle Nerve. 2018 Mar;57(3):478-486. doi: 10.1002/mus.25763. Epub 2017 Aug 23.
Mesenchymal stem cells (MSCs) can differentiate into various cell types.
In this study we investigated the potential of human tonsil-derived MSCs (T-MSCs) for neuromuscular regeneration in trembler-J (Tr-J) mice, a model for Charcot-Marie-Tooth disease type 1A (CMT1A).
T-MSCs differentiated toward skeletal myocytes with increased expression of skeletal muscle-related markers (including troponin I type 1, and myogenin), and the formation of myotubes in vitro. In-situ transplantation of T-MSC-derived myocytes (T-MSC myocytes) into the gastrocnemius muscle in Tr-J mice enhanced motor function, with recovery of compound muscle action potential amplitudes. Morphology of the sciatic nerve and skeletal muscle recovered without the formation of teratomas, and the expression levels of nerve growth factor and glial-cell-line-derived neurotrophic factor were increased significantly in T-MSC myocytes compared with T-MSCs in vitro.
Transplantation of T-MSC myocytes could enable neuromuscular regeneration in patients with CMT1A. Muscle Nerve 57: 478-486, 2018.
间充质干细胞（MSCs）可分化为多种细胞类型。
本研究旨在探讨人扁桃体来源的间充质干细胞（T-MSCs）在震颤麻痹-J（Tr-J）小鼠，即 1A 型腓骨肌萎缩症（CMT1A）模型中的神经肌肉再生潜能。
T-MSCs 向成肌细胞分化，骨骼肌相关标志物（包括肌钙蛋白 I 型和肌生成素）表达增加，并在体外形成肌管。将 T-MSC 衍生的肌细胞（T-MSC 肌细胞）原位移植到 Tr-J 小鼠的腓肠肌中，可增强运动功能，复合肌肉动作电位幅度恢复。坐骨神经和骨骼肌的形态学得到恢复，没有形成畸胎瘤，与体外 T-MSCs 相比，T-MSC 肌细胞中神经生长因子和胶质细胞系源性神经营养因子的表达水平显著增加。
T-MSC 肌细胞的移植可能使 CMT1A 患者实现神经肌肉再生。肌肉神经 57:478-486, 2018。
