Treatment of X-Linked Severe Combined Immunodeficiency by in Utero Transplantation of Paternal Bone Marrow

Abstract
Severe combined immunodeficiency is a congenital syndrome due to various genetic abnormalities that cause susceptibility to infection, failure to thrive, lymphoid hypoplasia, very low levels of T lymphocytes, and hypogammaglobulinemia.1,2 Untreated, the disorder is usually fatal within the first year of life. We report the successful treatment of a fetus with the X-linked variant of severe combined immunodeficiency by the in utero transplantation of paternal bone marrow that was enriched with hematopoietic cell progenitors.

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