De Santis M, De Luca C, Mappa I, Cesari E, Quattrocchi T, Spagnuolo T, Visconti D, Noia G, Caruso A
Telefono Rosso, Teratology Information Service, Department of Obstetrics and Gynaecology, Sacro Cuore Catholic University, Rome, Italy.
Minerva Ginecol. 2011 Aug;63(4):387-98.
Early evidence suggests that in-utero stem cell transplantation represents a new therapeutic strategy for different congenital disease. Moreover, gene therapy constitutes one of the most promising new approach to treat a wide spectrum of genetic disorders. It was shown that the fetus could represent an ideal recipient because of his immunologic early naiveté in gestation that reduces the risk of immunoreactions. Clinical experience in human fetus was performed in order to treat immunodeficiency and metabolic disorders, hemoglobinopathies and some other genetic diseases. Use of alternative source (i.e., cord blood, placenta, membrane, amniotic fluid, fetal tissue) of stem cell transplanted has been only one of the several strategies to improve donor cell advantages on host stem cell. The present review focused on the clinical use and therapeutic potential of in-utero stem cell transplantation, reporting the outcome of human cases treated and the limits of this therapy and possible future applications.
早期证据表明,宫内干细胞移植是治疗不同先天性疾病的一种新的治疗策略。此外,基因治疗是治疗广泛的遗传疾病最有前景的新方法之一。研究表明,胎儿可能是理想的受体,因为其在妊娠期的免疫幼稚状态可降低免疫反应的风险。为治疗免疫缺陷、代谢紊乱、血红蛋白病和其他一些遗传疾病,已开展了人类胎儿的临床实验。使用替代来源(如脐带血、胎盘、胎膜、羊水、胎儿组织)的干细胞进行移植,只是提高供体细胞相对于宿主干细胞优势的几种策略之一。本综述重点关注宫内干细胞移植的临床应用和治疗潜力,报告已治疗的人类病例的结果、该疗法的局限性以及可能的未来应用。