Division of Pediatric Surgery, University of California, San Francisco, San Francisco, CA, USA.
Division of Pediatric General, Thoracic and Fetal Surgery, University of Pennsylvania Perelman School of Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Semin Fetal Neonatal Med. 2017 Dec;22(6):410-414. doi: 10.1016/j.siny.2017.05.003. Epub 2017 Jun 12.
Advances in our understanding of stem cells, gene editing, prenatal imaging and fetal interventions have opened up new opportunities for the treatment of congenital diseases either through in-utero stem cell transplantation or in-utero gene therapy. Improvements in ultrasound-guided access to the fetal vasculature have also enhanced the safety and efficacy of cell delivery. The fetal environment offers accessible stem cell niches, localized cell populations with large proliferative potential, and an immune system that is able to acquire donor-specific tolerance. In-utero therapy seeks to take advantage of these factors and has the potential to cure diseases prior to the onset of symptoms, a strategy that offers substantial social and economic benefits. In this article, we examine previous studies in animal models as well as clinical attempts at in-utero therapy. We also discuss the barriers to successful in-utero therapy and future strategies for overcoming these obstacles.
我们对干细胞、基因编辑、产前成像和胎儿干预的理解的进步为治疗先天性疾病开辟了新的机会，无论是通过子宫内干细胞移植还是子宫内基因治疗。超声引导进入胎儿脉管系统的改进也提高了细胞输送的安全性和有效性。胎儿环境提供了易于接近的干细胞生态位、具有巨大增殖潜力的局部细胞群体，以及能够获得供体特异性耐受的免疫系统。子宫内治疗旨在利用这些因素，并有可能在症状出现之前治愈疾病，这种策略具有巨大的社会和经济效益。在本文中，我们检查了动物模型中的先前研究以及子宫内治疗的临床尝试。我们还讨论了成功进行子宫内治疗的障碍以及克服这些障碍的未来策略。
