Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

1995-10

AUTHORS

Donald B. Kohn, Kenneth I. Weinberg, Jan A. Nolta, Linda N. Heiss, Carl Lenarsky, Gay M. Crooks, Mary E. Hanley, Geralyn Annett, Judith S. Brooks, Anthony El-Khoureiy, Kim Lawrence, Susie Wells, Robert C. Moen, John Bastian, Debora E. Williams-Herman, Melissa Elder, Diane Wara, Thomas Bowen, Michael S. Hershfield, Craig A. Mullen, R. Michael Blaese, Robertson Parkman

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. More... »

PAGES

1017-1023

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/nm1095-1017

DOI

http://dx.doi.org/10.1038/nm1095-1017

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1013283766

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/7489356


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