Merianos Demetri, Heaton Todd, Flake Alan W
Children's Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Biol Blood Marrow Transplant. 2008 Jul;14(7):729-40. doi: 10.1016/j.bbmt.2008.02.012. Epub 2008 Apr 25.
In utero hematopoietic stem cell transplantation (IUHCT) is a potential therapeutic alternative to postnatal hematopoietic stem cell transplantation (HSCT) for congenital hematologic disorders that can be diagnosed early in gestation and can be cured by HSCT. The rationale is to take advantage of normal events during hematopoietic and immunologic ontogeny to facilitate allogeneic hematopoietic engraftment. Although the rationale remains compelling, IUHCT has not yet achieved its clinical potential. Achieving therapeutic levels of engraftment by IUHCT alone remains challenging. However, considerable experimental progress has been made toward the clinical strategy of using IUHCT to induce donor-specific tolerance to facilitate a relatively nontoxic postnatal HSCT. Because donor specific tolerance induction requires relatively minimal engraftment, this strategy may hold the key to broad clinical application of IUHCT in the near future.
宫内造血干细胞移植（IUHCT）是一种潜在的治疗选择，可替代出生后造血干细胞移植（HSCT）用于治疗先天性血液系统疾病，这些疾病在妊娠早期即可诊断，且可通过HSCT治愈。其基本原理是利用造血和免疫发育过程中的正常事件来促进异基因造血植入。尽管这一基本原理仍然令人信服，但IUHCT尚未发挥其临床潜力。仅通过IUHCT实现治疗水平的植入仍然具有挑战性。然而，在使用IUHCT诱导供体特异性耐受以促进相对无毒的出生后HSCT的临床策略方面已经取得了相当大的实验进展。由于供体特异性耐受诱导所需的植入相对较少，这一策略可能是IUHCT在不久的将来广泛临床应用的关键。
