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Chapter 20.2 - Fetal stem cell transplantation

Clinical potential

from Section 2 - Fetal disease

Published online by Cambridge University Press:  05 February 2013

Mark D. Kilby
Affiliation:
Department of Fetal Medicine, University of Birmingham
Anthony Johnson
Affiliation:
Baylor College of Medicine, Texas
Dick Oepkes
Affiliation:
Department of Obstetrics, Leiden University Medical Center
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Summary

Introduction

Over the past decades fetal medicine with fetal therapy has evolved as a new field within obstetrics. Different treatment strategies have been explored and one of those is fetal stem cell transplantation. The rational for in-utero stem cell transplantation is based on the assumptions that treatment before birth is preferable because the target disease is either lethal for the fetus or will result in early childhood morbidity, making postnatal therapy less efficacious. In-utero stem cell transplantation should potentially represent a major step forward in the management of patients with congenital, hematological, metabolic, and immunological disorders. Traditionally the early first and second trimester fetus has been described as preimmune, i.e., incapable of mounting an adaptive immune response to allogeneic cells or pathogens. The concept of in-utero transplantations (IUTs) aims to take advantage of the naïve immunological system, and consequently transplantations could potentially be carried out across histoincompatibility barriers and with no need for immunomodulation or cytoablation.

The human fetus was subjected to treatment attempts with stem cell transplantation in 1989 when Touraine et al. published their first case [1] of IUT in a human fetus affected by bare lymphocyte syndrome. Since then we know of several X-severe combined immunodeficiency disease (SCID) cases treated in utero with stem cells [2–4]. All these children survived and were chimeric at birth.

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