In utero hematopoietic cell transplantation—recent progress and the potential for clinical application

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. This review will discuss recent experimental progress toward overcoming the barriers to allogeneic engraftment and new therapeutic strategies that may hasten clinical application.