Abraham Rebecca, Vricella Luca, Hibino Narutoshi
Medical Scientist Training Program, University of Chicago, Chicago, IL, USA.
Section of Cardiac Surgery, Department of Surgery, University of Chicago, Chicago, IL, USA.
Transl Pediatr. 2023 Aug 30;12(8):1592-1600. doi: 10.21037/tp-23-127. Epub 2023 Aug 2.
Hypoplastic left heart syndrome (HLHS) is a deadly congenital heart disease that arises when the left ventricle and outflow tract fail to develop appropriately, inhibiting the adequate perfusion of the rest of the body. Historically, this disease has been treated via a series of surgeries that allows the heart to use a single ventricle. These surgeries are often a palliative measure, and heart transplantation is the only definitive therapy that exists for this condition. It has been hypothesized that stem cell-based regenerative therapies could have a role in promoting cardiac tissue regeneration in HLHS patients who are undergoing palliative surgery. Several clinical trials have demonstrated that introducing pluripotent cells into the heart is safe, feasible, and capable of improving right ventricular ejection fraction (RVEF). However, while these approaches show great promise, there is still room for development. There is a substantial body of pre-clinical work that is focused on generating increasingly large and complex pieces of cardiac tissue in the form of cardiac patches, with the idea that these could be used to rebuild and strengthen the heart in a robust and long-lasting manner. In total, stem cell-based therapies have much to offer when it comes to improving the treatment of HLHS.
左心发育不全综合征(HLHS)是一种致命的先天性心脏病,当左心室和流出道发育异常时就会出现,从而抑制了身体其他部位的充分灌注。从历史上看,这种疾病一直通过一系列手术进行治疗,使心脏能够使用单一心室。这些手术通常是一种姑息性措施,而心脏移植是针对这种病症唯一存在的确定性治疗方法。据推测,基于干细胞的再生疗法可能在促进接受姑息性手术的HLHS患者的心脏组织再生中发挥作用。多项临床试验表明,将多能细胞引入心脏是安全、可行的,并且能够提高右心室射血分数(RVEF)。然而,尽管这些方法显示出巨大的前景,但仍有发展空间。有大量临床前研究工作致力于以心脏补片的形式生成越来越大且复杂的心脏组织块,其理念是这些组织块可用于以强大而持久的方式重建和强化心脏。总体而言,在改善HLHS的治疗方面,基于干细胞的疗法有很多可提供的。