Ando Yu, Saito Marie, Machida Masakazu, Yoshida-Noro Chikako, Akutsu Hidenori, Takahashi Masataka, Toyoda Masashi, Umezawa Akihiro
Center for Regenerative Medicine, National Research Institute for Child Health and Development, Tokyo 157-8535, Japan.
Department of Applied Molecular Chemistry, College of Industrial Technology, Nihon University, Tokyo, Japan.
Stem Cells Int. 2017;2017:7541734. doi: 10.1155/2017/7541734. Epub 2017 Jun 15.
A large number of myocytes are necessary to treat intractable muscular disorders such as Duchenne muscular dystrophy with cell-based therapies. However, starting materials for cellular therapy products such as myoblasts, marrow stromal cells, menstrual blood-derived cells, and placenta-derived cells have a limited lifespan and cease to proliferate in vitro. From the viewpoints of manufacturing and quality control, cells with a long lifespan are more suitable as a starting material. In this study, we generated stromal cells for future myoblast therapy from a working cell bank of human embryonic stem cells (ESCs). The ESC-derived CD105 cells with extensive in vitro proliferation capability exhibited myogenesis and genetic stability in vitro. These results imply that ESC-derived CD105 cells are another cell source for myoblasts in cell-based therapy for patients with genetic muscular disorders. Since ESCs are immortal, mesenchymal stromal cells generated from ESCs can be manufactured at a large scale in one lot for pharmaceutical purposes.
对于采用基于细胞的疗法治疗诸如杜氏肌营养不良症等难治性肌肉疾病而言，需要大量的肌细胞。然而，细胞治疗产品的起始材料，如成肌细胞、骨髓基质细胞、月经血来源细胞和胎盘来源细胞，其寿命有限，在体外会停止增殖。从生产制造和质量控制的角度来看，寿命长的细胞更适合作为起始材料。在本研究中，我们从人胚胎干细胞（ESC）的工作细胞库中生成了用于未来成肌细胞治疗的基质细胞。具有广泛体外增殖能力的ESC衍生的CD105细胞在体外表现出成肌作用和遗传稳定性。这些结果表明，ESC衍生的CD105细胞是基于细胞的疗法治疗遗传性肌肉疾病患者时成肌细胞的另一种细胞来源。由于ESC是永生的，从ESC生成的间充质基质细胞可大规模批量生产用于制药目的。
