Treatment of X-Linked Severe Combined Immunodeficiency by in Utero Transplantation of Paternal Bone Marrow
Authors:
- Maria-Grazia RoncaroloMRMaria-Grazia Roncarolo
- Jennifer M. PuckJPJennifer M. Puck
- Graza Almeida-PoradaGAGraza Almeida-Porada
- Mark I. EvansMEMark I. Evans
- Mark P. JohnsonMJMark P. Johnson
- Estaban M. AbellaEAEstaban M. Abella
- Duane D. HarrisonDHDuane D. Harrison
- Esmail D. ZanjaniEZEsmail D. Zanjani
- 12 December 1996
- journal article
- research article
- Published by Massachusetts Medical Society in New England Journal of Medicine
- Vol. 335 (24) , 1806-1810
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.Keywords
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