Perinatal fetal-cell and gene therapy

Several inherited diseases can now be treated using postnatal or prenatal, in utero, transplantation of stem cells from the human fetal liver. Twenty- four patients with severe immunodeficiency diseases have been treated in in fancy and three at the fetal stage, in our institution. We have also treate d similarly 34 patients with inborn errors of metabolism or hemoglobinopath ies. A total of 64% of all patients are alive with either full cure of thei r initial disease or at least significant benefit from the treatment. Immun ological reconstitution can develop despite full mismatch between the stem cell donor and the recipient patient. Tolerance is acquired both towards do nor and host antigens. Gene therapy is starting its development in some inf ants with an immunodeficiency related to a known gene defect and, in the fu ture, it may be used in fetuses, immediately after the prenatal diagnosis o f the gene abnormality. (C) 2000 International Society for Immunopharmacolo gy. Published by Elsevier Science Ltd. All rights reserved.