[HTML][HTML] Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow

AW Flake, MG Roncarolo, JM Puck… - … England Journal of …, 1996 - Mass Medical Soc
AW Flake, MG Roncarolo, JM Puck, G Almeida-Porada, MI Evans, MP Johnson, EM Abella…
New England Journal of Medicine, 1996Mass Medical Soc
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. Case Report …
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.
Case Report
The patient, 11 months old at this writing, is the second son of a 28-year-old woman known to carry a mutation found in . . .
The New England Journal Of Medicine