title: In utero hematopoietic cell transplantation: what are the important questions?
abstract: The treatment of congenital hematologic disorders before birth by in utero hematopoietic stem cell transplantation remains a challenging goal. Although success has been achieved in X-linked severe combined immunodeficiency, the approach has failed in all other disorders attempted thus far. In this review, we examine relevant experimental data from the perspective of an analysis of why failure has occurred. We will also attempt to pose the important questions that will need to be answered prior to further clinical attempts to treat most target disorders by this approach.
authors.affiliation: Children's Institute of Surgical Science, Children's Hospital of Philadelphia, Philadelphia, Pa. 19104-4318, USA.
authors.forename: William H
authors.initials: WH
authors.lastname: Peranteau
authors.forename: Satoshi
authors.initials: S
authors.lastname: Hayashi
authors.forename: Heung Bae
authors.initials: HB
authors.lastname: Kim
authors.forename: Aimen F
authors.initials: AF
authors.lastname: Shaaban
authors.forename: Alan W
authors.initials: AW
authors.lastname: Flake
mesh_terms: D005260:Female; D005333:Fetus / Q000276:immunology / Q000601:surgery*; D006085:Graft Survival; D006402:Hematologic Diseases / Q000151:congenital / Q000601:surgery; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D011247:Pregnancy