Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

D. B. Kohn, K. I. Weinberg, Jan Nolta, L. N. Heiss, C. Lenarsky, G. M. Crooks, M. E. Hanley, G. Annett, J. S. Brooks, A. El-Khoureiy, K. Lawrence, S. Wells, R. C. Moen, J. Bastian, D. E. Williams-Herman, M. Elder, D. Wara, T. Bowen, M. S. Hershfield

Research output: Contribution to journalArticle

461 Scopus citations

Abstract

Haematopoietic stem cells in umbilical cord blood are an attractive target for gene therapy of inborn errors of metabolism. Three neonates with severe combined immunodeficiency were treated by retroviral-mediated transduction of the CD34+ cells from their umbilical cord blood with a normal human adenosine deaminase complementary DNA followed by autologous transplantation. The continued presence and expression of the introduced gene in leukocytes from bone marrow and peripheral blood for 18 months demonstrates that umbilical cord blood cells may be genetically modified with retroviral vectors and engrafted in neonates for gene therapy.

Original languageEnglish (US)
Pages (from-to)1017-1023
Number of pages7
JournalNature Medicine
Volume1
Issue number10
StatePublished - 1995
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)

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    Kohn, D. B., Weinberg, K. I., Nolta, J., Heiss, L. N., Lenarsky, C., Crooks, G. M., Hanley, M. E., Annett, G., Brooks, J. S., El-Khoureiy, A., Lawrence, K., Wells, S., Moen, R. C., Bastian, J., Williams-Herman, D. E., Elder, M., Wara, D., Bowen, T., & Hershfield, M. S. (1995). Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Medicine, 1(10), 1017-1023.