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诱导多能干细胞在先天性心脏病研究与治疗中的变革潜力。

Transformative Potential of Induced Pluripotent Stem Cells in Congenital Heart Disease Research and Treatment.

作者信息

Mashali Mohammed A, Deschênes Isabelle, Saad Nancy S

机构信息

Department of Physiology and Cell Biology, Dorothy M. Davis Heart and Lung Research Institute, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.

Department of Surgery, Faculty of Veterinary Medicine, Damanhour University, Damanhour 22514, Egypt.

出版信息

Children (Basel). 2025 May 23;12(6):669. doi: 10.3390/children12060669.

Abstract

Congenital heart disease (CHD), the most common congenital anomaly, remains a significant lifelong burden despite advancements in medical and surgical interventions. Induced pluripotent stem cells (iPSCs) have emerged as a groundbreaking platform in CHD research, offering patient-specific models to investigate the genetic, epigenetic, and molecular mechanisms driving the disease. Utilizing technologies such as CRISPR/Cas9 gene editing, cardiac organoids, and high-throughput screening, iPSCs enable innovative strategies in disease modeling, precision drug discovery, and regenerative therapies. However, clinical translation faces challenges related to immaturity, differentiation variability, large-scale feasibility, and tumorigenicity. Addressing these barriers will require standardized protocols, bioengineering solutions, and interdisciplinary collaboration. This review examines the critical role of iPSCs in advancing CHD research and care, demonstrating their potential to revolutionize treatment through patient-specific, regenerative approaches. By addressing current limitations and advancing iPSC technology, the field is positioned to pave the way for precision-based CHD therapies for this lifelong condition.

摘要

先天性心脏病(CHD)是最常见的先天性畸形,尽管医学和外科干预取得了进展,但它仍然是一个重大的终身负担。诱导多能干细胞(iPSC)已成为CHD研究中的一个开创性平台,提供了患者特异性模型来研究驱动该疾病的遗传、表观遗传和分子机制。利用CRISPR/Cas9基因编辑、心脏类器官和高通量筛选等技术,iPSC在疾病建模、精准药物发现和再生疗法方面实现了创新策略。然而,临床转化面临着与不成熟、分化变异性、大规模可行性和致瘤性相关的挑战。解决这些障碍需要标准化方案、生物工程解决方案和跨学科合作。本综述探讨了iPSC在推进CHD研究和治疗中的关键作用,展示了它们通过患者特异性、再生方法彻底改变治疗的潜力。通过解决当前的局限性并推进iPSC技术,该领域有望为这种终身疾病的精准CHD疗法铺平道路。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3750/12190934/55b378ff9402/children-12-00669-g001.jpg

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