Chowdhury Neeladri, Asakura Atsushi
Stem Cell Institute, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Paul and Sheila Wellstone Muscular Dystrophy Center, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
Stem Cells Int. 2017;2017:3027520. doi: 10.1155/2017/3027520. Epub 2017 May 15.
Muscular dystrophies, myopathies, and traumatic muscle injury and loss encompass a large group of conditions that currently have no cure. Myoblast transplantations have been investigated as potential cures for these conditions for decades. However, current techniques lack the ability to generate cell numbers required to produce any therapeutic benefit. In utero stem cell transplantation into embryos has been studied for many years mainly in the context of hematopoietic cells and has shown to have experimental advantages and therapeutic applications. Moreover, patient-derived cells can be used for experimental transplantation into nonhuman animal embryos via in utero injection as the immune response is absent at such early stages of development. We therefore propose in utero transplantation as a potential method to generate patient-derived humanized skeletal muscle as well as muscle stem cells in animals for therapeutic purposes as well as patient-specific drug screening.
肌肉萎缩症、肌病以及创伤性肌肉损伤和缺失涵盖了一大类目前无法治愈的病症。几十年来，成肌细胞移植一直被作为这些病症的潜在治疗方法进行研究。然而，目前的技术缺乏产生具有任何治疗益处所需细胞数量的能力。子宫内干细胞移植到胚胎中已经研究多年，主要是在造血细胞的背景下进行的，并且已显示出具有实验优势和治疗应用。此外，患者来源的细胞可通过子宫内注射用于实验性移植到非人类动物胚胎中，因为在发育的早期阶段不存在免疫反应。因此，我们提出子宫内移植作为一种潜在的方法，用于在动物体内生成患者来源的人源化骨骼肌以及肌肉干细胞，用于治疗目的以及患者特异性药物筛选。
