In Utero Stem Cell Transplantation

Autor: Michael R. Harrison, Esmail D. Zanjani, Alan W. Flake
Rok vydání: 2016
Předmět:
Zdroj: Fetal Stem Cells in Regenerative Medicine ISBN: 9781493934812
DOI: 10.1007/978-1-4939-3483-6_17
Popis: During the last few decades, the human fetus has become a candidate for therapeutic interventions; however, most of these therapies are surgical in nature and target specific anatomic defects that benefit from prenatal intervention. In this chapter we review the rationale for cell-based in utero therapy, namely in utero hematopoietic stem cell transplantation (IUHCT). The primary rationale for IUHCT is that early introduction of cells into the pre-immune fetus can take advantage of normal hematopoietic and immunologic development, leading to allogeneic engraftment. IUHCT may offer a prenatal cure to many congenital hematopoietic diseases and be a safe, more effective alternative to postnatal hematopoietic stem cell transplantation (HSCT). While there are various barriers to IUHCT and successful donor cell engraftment, enormous progress has been made in validating this therapy in both small and large animal models. We will review the recent improvements in technique, timing, safety, donor graft content, and manipulations targeting donor cells and the host hematopoietic compartment. Many of the former barriers to IUHCT have been elucidated and surmounted, bringing us to the point at which a clinical trial may be justified.
Databáze: OpenAIRE