Surbek D V, Holzgreve W
Universitäts-Frauenklinik, Basel.
Ther Umsch. 2002 Nov;59(11):619-23. doi: 10.1024/0040-5930.59.11.619.
Prenatal stem cell transplantation is a novel, promising therapeutic option for genetic disorders, which is now at the edge of moving from preclinical research into clinical application. The first clinical experience shows that inborn diseases, which lead to a severe immunodeficiency, can be treated successfully inutero. No therapeutic success has been achieved in genetic disorders which do not severely affect the immune system. Therefore, new strategies to improve the success are being developed, including e.g., graft modification, prenatal conditioning of the fetus, postnatal re-transplantation after prenatal induction of immune tolerance, and fetal gene therapy using autologous fetal stem cells. The use of non-hematopoietic (e.g. mesenchymal) or pluripotent stem cells will most probably lead to an expansion of the spectrum of indications in genetic diseases for this novel treatment. At the same time, however, ethical implications, in particular regarding fetal gene therapy and the use of pluripotent stem cells must be evaluated.
产前干细胞移植是一种针对遗传疾病的新型、有前景的治疗选择,目前正处于从临床前研究迈向临床应用的边缘。首例临床经验表明,导致严重免疫缺陷的先天性疾病可在子宫内成功治疗。对于未严重影响免疫系统的遗传疾病,尚未取得治疗成功。因此,正在开发新的策略以提高成功率,包括例如移植物修饰、胎儿的产前预处理、产前诱导免疫耐受后的产后再次移植以及使用自体胎儿干细胞的胎儿基因治疗。使用非造血(如间充质)或多能干细胞很可能会扩大这种新型治疗在遗传疾病中的适应症范围。然而,与此同时,必须评估伦理影响,特别是关于胎儿基因治疗和多能干细胞的使用。