基于人类诱导多能干细胞的先天性心脏病模型及治疗方法的当前进展。

Current advances in human-induced pluripotent stem cell-based models and therapeutic approaches for congenital heart disease.

作者信息

Cao Meiling, Liu Yanshan, Sun Ying, Han Ruiyi, Jiang Hongkun

机构信息

Department of Neonatology, The First Hospital of China Medical University, Shenyang, 110001, Liaoning, China.

Department of Pediatrics, The First Hospital of China Medical University, No.155 Nanjing North Street, Heping District, Shenyang, 110001, Liaoning, China.

出版信息

Mol Cell Biochem. 2025 Jan;480(1):159-172. doi: 10.1007/s11010-024-04997-z. Epub 2024 Apr 18.

Abstract

Congenital heart disease (CHD) represents a significant risk factor with profound implications for neonatal survival rates and the overall well-being of adult patients. The emergence of induced pluripotent stem cells (iPSCs) and their derived cells, combined with CRISPR technology, high-throughput experimental techniques, and organoid technology, which are better suited to contemporary research demands, offer new possibilities for treating CHD. Prior investigations have indicated that the paracrine effect of exosomes may hold potential solutions for therapeutic intervention. This review provides a summary of the advancements in iPSC-based models and clinical trials associated with CHD while elucidating potential therapeutic mechanisms and delineating clinical constraints pertinent to iPSC-based therapy, thereby offering valuable insights for further deliberation.

摘要

先天性心脏病(CHD)是一个重要的风险因素,对新生儿存活率和成年患者的整体健康有着深远影响。诱导多能干细胞(iPSC)及其衍生细胞的出现,与更适合当代研究需求的CRISPR技术、高通量实验技术和类器官技术相结合,为治疗CHD提供了新的可能性。先前的研究表明,外泌体的旁分泌作用可能为治疗干预提供潜在的解决方案。本综述总结了基于iPSC的模型和与CHD相关的临床试验的进展,同时阐明了潜在的治疗机制,并描述了与基于iPSC的治疗相关的临床限制,从而为进一步的思考提供有价值的见解。

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