摘要
晚期心脏衰竭是一种进展性顽固性疾病,它是导致死亡的主要原因。功能性心肌细胞的补充疗法表现为心肌细胞显著减少,因此该方法是一种理想的治疗方案。最近,科学家建立了利用诱导多能干细胞(iPSC)技术代替使用人类胚胎无限体外生产的人功能性心肌细胞的技术。一系列的基础研究表明,将ipsc衍生而来的心肌细胞移植入受损心脏能使心脏功能恢复,从而建立iPSC移植治疗的“概念证明”理论。然而,考虑到该治疗方法的临床应用,在临床前阶段,其可行性、安全性和治疗效果需要得到进一步研究。本文总结了与iPSC-CMs移植治疗心脏疾病的安全性和治疗效果相关的最新研究并讨论了其临床研究的前景。
关键词: iPSC,心肌细胞,移植,免心脏衰竭,疫原性,心肌成形。
Current Gene Therapy
Title:Building A New Treatment For Heart Failure-Transplantation of Induced Pluripotent Stem Cell-derived Cells into the Heart
Volume: 16 Issue: 1
Author(s): Shigeru Miyagawa, Satsuki Fukushima, Yukiko Imanishi, Takuji Kawamura and Noriko Mochizuki- Oda, Shigeo Masuda and Yoshiki Sawa
Affiliation:
关键词: iPSC,心肌细胞,移植,免心脏衰竭,疫原性,心肌成形。
摘要: Advanced cardiac failure is a progressive intractable disease and is the main cause of mortality and morbidity worldwide. Since this pathology is represented by a definite decrease in cardiomyocyte number, supplementation of functional cardiomyocytes into the heart would hypothetically be an ideal therapeutic option. Recently, unlimited in vitro production of human functional cardiomyocytes was established by using induced pluripotent stem cell (iPSC) technology, which avoids the use of human embryos. A number of basic studies including ours have shown that transplantation of iPSCderived cardiomyocytes (iPSC-CMs) into the damaged heart leads to recovery of cardiac function, thereby establishing “proof-of-concept” of this iPSC-transplantation therapy. However, considering clinical application of this therapy, its feasibility, safety, and therapeutic efficacy need to be further investigated in the pre-clinical stage. This review summarizes up-to-date important topics related to safety and efficacy of iPSC-CMs transplantation therapy for cardiac disease and discusses the prospects for this treatment in clinical studies.
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Shigeru Miyagawa, Satsuki Fukushima, Yukiko Imanishi, Takuji Kawamura and Noriko Mochizuki- Oda, Shigeo Masuda and Yoshiki Sawa , Building A New Treatment For Heart Failure-Transplantation of Induced Pluripotent Stem Cell-derived Cells into the Heart, Current Gene Therapy 2016; 16 (1) . https://dx.doi.org/10.2174/1566523216666160119094143
DOI https://dx.doi.org/10.2174/1566523216666160119094143 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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