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Keeping your strength up: induced pluripotent stem cell-based approaches for the treatment and investigation of skeletal muscle disorders
Frontiers in Life Science ( IF 1.333 ) Pub Date : 2023-05-05 , DOI: 10.1080/26895293.2023.2207774
Hyun-Jun Kim 1 , Da-Woon Jung 1 , Darren R. Williams 1
Affiliation  

Skeletal muscle comprises the major organ system in our bodies and is the effector tissue for fundamental processes, such as movement, breathing and glucose homeostasis. However, muscle regeneration can be insufficient to repair major injuries or become exhausted by the ongoing fiber damage that occurs in muscular dystrophy. Cell therapy to provide a source of myogenic stem cells that repopulate the lost muscle fibers is an attractive option to treat skeletal muscle injury or dystrophy. Induced pluripotent stem cells (iPSCs) have the potential to be an ideal source of donor muscle cells. This review will discuss major research applications of iPSCs to facilitate skeletal muscle repair, which involve myogenic cell transplantation, in vitro modeling of normal and dystrophic muscle tissue, and drug screening protocols. Recent advances in these fields will be discussed in this review, such as the production of neuromuscular junctions between muscle fibers and motor neurons, along with an overview of the problems that currently prevent iPSCs from being a clinically valid source of transplantable skeletal muscle stem cells. Potential future applications of iPSCs to enhance skeletal muscle regeneration will also be discussed.



中文翻译:

保持体力:基于诱导多能干细胞的方法治疗和研究骨骼肌疾病

骨骼肌是我们身体的主要器官系统,是运动、呼吸和葡萄糖稳态等基本过程的效应组织。然而,肌肉再生可能不足以修复重大损伤或因肌营养不良症中发生的持续纤维损伤而变得疲惫不堪。提供生肌干细胞来源的细胞疗法可以重新填充丢失的肌纤维,是治疗骨骼肌损伤或营养不良的一个有吸引力的选择。诱导多能干细胞 (iPSC) 有可能成为供体肌肉细胞的理想来源。本综述将讨论 iPSC 在促进骨骼肌修复方面的主要研究应用,包括生肌细胞移植、正常和营养不良肌肉组织的体外建模以及药物筛选方案。本综述将讨论这些领域的最新进展,例如肌肉纤维和运动神经元之间神经肌肉接头的产生,以及目前阻碍 iPSC 成为临床上有效的可移植骨骼肌干细胞来源的问题的概述。还将讨论 iPSC 在增强骨骼肌再生方面的潜在未来应用。

更新日期:2023-05-05
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