Cell therapy for duchenne muscular dystrophy using induced pluripotent stem cell-derived muscle stem cells and the potential of regenerative rehabilitation

IF 4.7 3区 工程技术 Q2 ENGINEERING, BIOMEDICAL
Nana Takenaka-Ninagawa , Megumi Goto , Clémence Kiho Bourgeois Yoshioka , Mayuho Miki , Hidetoshi Sakurai
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引用次数: 0

Abstract

Duchenne muscular dystrophy (DMD) is a hereditary disease characterized by severe muscle weakness resulting from DYSTROPHIN deficiency-associated damage. Muscle regeneration therapy using skeletal muscle stem cell (MuSC) transplantation has demonstrated great promise. Optimized differentiation methods yield efficient induced pluripotent stem cell-derived MuSCs (iMuSCs); transplanted iMuSCs have high regenerative potential in DMD mouse models. However, achieving complete pathophysiological recovery of DMD motor function through cell therapy alone is challenging. According to recent studies, exercise can significantly improve DMD pathophysiology. Current research aims to enhance therapeutic efficacy by combining cell transplantation with exercise interventions, addressing the limitations of cell-transplantation therapy for DMD.

利用诱导多能干细胞衍生的肌肉干细胞治疗杜兴氏肌肉萎缩症的细胞疗法和再生康复的潜力
杜兴氏肌营养不良症(DMD)是一种遗传性疾病,其特征是因DYSTROPHIN缺乏症相关损伤而导致的重症肌无力。利用骨骼肌干细胞(MuSC)移植进行肌肉再生治疗已显示出巨大前景。经过优化的分化方法产生了高效的诱导多能干细胞衍生的MuSCs(iMuSCs);移植的iMuSCs在DMD小鼠模型中具有很高的再生潜力。然而,仅通过细胞疗法实现 DMD 运动功能的完全病理生理恢复具有挑战性。根据最近的研究,运动可以显著改善 DMD 的病理生理学。目前的研究旨在通过将细胞移植与运动干预相结合来提高疗效,从而解决细胞移植治疗 DMD 的局限性。
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来源期刊
Current Opinion in Biomedical Engineering
Current Opinion in Biomedical Engineering Medicine-Medicine (miscellaneous)
CiteScore
8.60
自引率
2.60%
发文量
59
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