ENGRAFTMENT OF GENE-MODIFIED UMBILICAL-CORD BLOOD-CELLS IN NEONATES WITH ADENOSINE-DEAMINASE DEFICIENCY

Haematopoietic stem cells in umbilical cord blood are an attractive ta rget for gene therapy of inborn errors of metabolism. Three neonates w ith severe combined immunodeficiency were treated by retroviral-mediat ed transduction of the CD34+ cells from their umbilical cord blood wit h a normal human adenosine deaminase complementary DNA followed by aut ologous transplantation. The continued presence and expression of the introduced gene in leukocytes from bone marrow and peripheral blood fo r 18 months demonstrates that umbilical cord blood cells may be geneti cally modified with retroviral vectors and engrafted in neonates for g ene therapy.