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宫内造血干细胞移植(IUHCT)和宫内基因治疗(IUGT)对一些在胎儿期或出生后早期已造成不可逆损害的单基因遗传病显示了潜在的治疗前景,但仍存在很多问题。IUHCT后宿主造血区的容受性、宿主造血细胞的竞争、植入移植物的免疫屏障是有待解决的难题。通过IUHCT诱导供体特异性的移植物免疫耐受,出生后进行无毒性(或低毒性)的骨髓移植,是目前有临床前景的治疗策略。IUGT仍处于早期研究阶段,有关基因插入突变、影响器官发育、低水平的生殖细胞传递等安全性问题仍然需要深入研究。此外,必须考虑到IUGT的伦理学和潜在改变人类基因组的问题。
Intrauterine hematopoietic stem cell transplantation (IUHCT) and intrauterine gene therapy (IUGT) have shown potential therapeutic prospects for some single-gene genetic diseases that have caused irreversible damage during the fetal or early postnatal period, but many problems remain. The acceptability of host hematopoietic region after IUHCT, the competition of host hematopoietic cells, and the implantation of the immune barrier of the graft are problems to be solved. It is currently a clinically promising strategy to induce donor-specific graft immune tolerance through IUHCT and to develop a non-toxic (or low toxicity) bone marrow transplant after birth. IUGT is still in the early stage of research, and further studies are needed on the safety issues such as gene mutation, organ development and low level of germ cell transmission. In addition, IUGT’s ethics and potential changes to the human genome must be considered.