In utero stem cell transplantation: two steps forward but one step back?

Abstract

The concept of in utero stem cell transplantation has provided hope that at least some of the genetic disorders which can be diagnosed prenatally can be treated before the pathological sequelae become manifested. Our increasing knowledge of haematopoietic stem cell biology has provided further possibilities for optimising this transplant procedure. However, the relatively poor success rate achieved using this procedure has prompted some to suggest that there may be fetal rejection of donor cells via an immune mechanism. However, the prevailing evidence from both animal models and from human cases, suggest that there may be other mechanisms which are preventing successful engraftment in some disorders and these must be addressed if the procedure is to receive further attention. The ability to achieve sustained engraftment after in utero transplantation will have relevance for those seeking to use gene therapy as an alternative therapeutic stratagem.

• fetal haematopoiesis
• in utero transplantation
• stem cells
• tolerance