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Stem Cell Therapy and Congenital Heart Disease

机译:干细胞疗法与先天性心脏病

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For more than a decade, stem cell therapy has been the focus of intensive efforts for the treatment of adult heart disease, and now has promise for treating the pediatric population. On the basis of encouraging results in the adult field, the application of stem cell-based strategies in children with congenital heart disease (CHD) opens a new therapy paradigm. To date, the safety and efficacy of stem cell-based products to promote cardiac repair and recovery in dilated cardiomyopathy and structural heart disease in infants have been primarily demonstrated in scattered clinical case reports, and supported by a few relevant pre-clinical models. Recently the TICAP trial has shown the safety and feasibility of intracoronary infusion of autologous cardiosphere-derived cells in children with hypoplastic left heart syndrome. A focus on preemptive cardiac regeneration in the pediatric setting may offer new insights as to the timing of surgery, location of cell-based delivery, and type of cell-based regeneration that could further inform acquired cardiac disease applications. Here, we review the current knowledge on the field of stem cell therapy and tissue engineering in children with CHD, and discuss the gaps and future perspectives on cell-based strategies to treat patients with CHD.
机译:十多年来,干细胞疗法一直是治疗成人心脏病的重点工作,现在有望用于治疗儿科人群。在成人领域取得令人鼓舞的成果的基础上,基于干细胞的策略在先天性心脏病(CHD)儿童中的应用开辟了新的治疗范例。迄今为止,以干细胞为基础的产品促进婴儿扩张型心肌病和结构性心脏病的心脏修复和恢复的安全性和有效性主要在分散的临床病例报告中得到了证明,并得到了一些相关的临床前模型的支持。最近,TICAP试验表明,在患有发育不良的左心综合征的儿童中,冠状动脉内灌注自体心球来源的细胞的安全性和可行性。儿科环境中先发制人的心脏再生的重点可能会提供有关手术时机,基于细胞的分娩位置和基于细胞的再生类型的新见解,这些信息可能会进一步为获得性心脏病的治疗提供依据。在这里,我们回顾了儿童冠心病干细胞治疗和组织工程领域的最新知识,并讨论了基于细胞的策略来治疗冠心病的差距和未来的前景。

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