Molecular Approaches in Single Ventricle Management.
Molecular Approaches in Single Ventricle Management.
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DOI：
10.1053/j.pcsu.2020.03.003
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发表时间：
2020
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在过去的20年里，医学和外科治疗的进步显著改善了单心室先天性心脏病的早期预后。尽管取得了这些进展，但长期结果仍然不理想，即使在现代，解决系统性心室和/或Fontan衰竭的治疗选择也很有限。复杂的分子生物学技术揭示了单心室疾病的发展机制。人们正在努力利用这些知识来改善临床诊断、治疗和预后。旨在诱导心肌细胞增殖和预防迟发性心功能障碍的细胞疗法已经进入临床领域。在不久的将来，一些新的生物疗法有望用于单心室疾病患者。这些科学进步为我们提供了希望，并重申了我们的信念，即分子医学将迎来新一代的单心室治疗方法。
Advances in medical and surgical management have significantly improved early outcomes in single ventricle congenital heart disease over the last 2 decades. Despite these advances, long-term outcomes remain suboptimal and therapeutic options to address systemic ventricular and/or Fontan failure are limited even in the modern era. Intricate molecular biologic techniques have shed light into the mechanisms of development of single ventricle disease. Efforts are underway to leverage this knowledge to improve clinical diagnosis, therapy, and prognostication. Cell-based therapies aimed at inducing cardiomyocyte proliferation and preventing delayed cardiac dysfunction have already entered the clinical realm. Several more novel biological therapies are expected to become available for patients with single ventricle disease in the near future. These scientific advancements provide us hope and reaffirm our faith that molecular medicine will usher in the next generation of therapies for single ventricle management.