28 July 1997

Physicians at the Cedars-Sinai Medical Center and the Childrens Hospital Los Angeles are investigating the efficacy of in utero bone marrow transplantation to treat genetic diseases involving hematopoietic stem cells. CD34+ cells isolated from the father's bone marrow will be transplanted initially during the first trimester, i.e. before 14 weeks of gestation. Potential candidates are fetuses whose diagnosis is confirmed before 11 weeks of gestation and who do not have an identifiable histocompatible family member. Candidate diseases include all forms of severe combine immune deficiency, Wiskott-Aldrich syndrome, chronic granulomatous disease, a thalassemia, Hurler syndrome, metachromatic leukodystrophy and Krabbe disease.
This project has been approved by both the FDA and the Institutional Review Board (IRB) at each of the participating sites, including Cedars-Sinai Medical Center and Childrens Hospital Los Angeles.