Child Health and Development Institute, Mount Sinai School of Medicine, One Gustave Levy Place, New York, NY 10029, USA.
Curr Opin Cardiol. 2011 May;26(3):223-9. doi: 10.1097/HCO.0b013e32834598ad.
The development of induced pluripotent stem cell (iPSC) technology has led to many advances in the areas of directed cell differentiation and characterization. New methods for generating iPSC-derived cardiomyocytes provide an invaluable resource for the study of certain cardiovascular disorders. This review highlights the current technology in this field, its application thus far to the study of genetic disorders of the RAS/MAPK pathway and long-QT syndrome (LQTS), and future directions for the field.
Enhanced methods increase the efficiency of generating and stringently purifying iPSC-derived cardiomyocytes. The use of cardiomyocytes derived from patients with LEOPARD syndrome and LQTS has shed light on the molecular mechanisms of disease and validated their use as reliable human disease models.
The use of iPSC-derived cardiomyocytes to study genetic cardiovascular disorders will enable a deeper and more applicable understanding of the molecular mechanisms of human disease, as well as improving our ability to achieve successful cell-based therapies. Methods to efficiently generate these cells are improving and provide promise for future applications of this technology.
目的综述：诱导多能干细胞（iPSC）技术的发展推动了定向细胞分化和鉴定领域的许多进展。生成 iPSC 衍生心肌细胞的新方法为研究某些心血管疾病提供了宝贵的资源。本综述强调了该领域的当前技术，迄今为止其在 RAS/MAPK 通路和长 QT 综合征（LQTS）遗传疾病研究中的应用，以及该领域的未来方向。
最新发现：增强的方法提高了生成和严格纯化 iPSC 衍生心肌细胞的效率。使用来自 LEOPARD 综合征和 LQTS 患者的心肌细胞阐明了疾病的分子机制，并验证了它们作为可靠的人类疾病模型的用途。
总结：使用 iPSC 衍生的心肌细胞研究遗传心血管疾病将使我们能够更深入地了解人类疾病的分子机制，并提高我们实现成功细胞治疗的能力。生成这些细胞的有效方法正在不断改进，并为该技术的未来应用提供了前景。
