A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34+ cells from the bone marrow of human immunodeficiency virus-1-infected children

Donald B. Kohn, Gerhard Bauer, C. Robert Rice, J. C. Rothschild, Denise A. Carbonaro, Penelope Valdez, Qian Lin Hao, Chen Zhou, Ingrid Bahner, Karen Kearns, Kate Brody, Sarah Fox, Elizabeth Haden, Kathy Wilson, Cathy Salata, Cathy Dolan, Charles Wetter, Estuardo Aguilar-Cordova, Joseph Church

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Abstract

Genetic modification of hematopoietic stem cells with genes that inhibit replication of human immunodeficiency virus:1 (HIV-1) could lead to development of T lymphocytes and monocytic cells resistant to HIV-1 infection after transplantation. We performed a clinical trial to evaluate the safety and feasibility of this procedure, using bone marrow from four HIV-1-infected pediatric subjects (ages 8 to 17 years). We obtained bone marrow, isolated CD34+ cells, performed in vitro transduction with a retroviral vector carrying a rev-responsive element (RRE) decoy gene, and reinfused the cells into these subjects with no evidence of adverse effects. The levels of gene- containing leukocytes in peripheral blood samples in the I year after gene transfer/cell infusion have been extremely low. These observations support the potential of performing gene therapy for HIV-1 using hematopoietic cells, but emphasize the need for improved gene transfer techniques.

Original languageEnglish (US)
Pages (from-to)368-371
Number of pages4
JournalBlood
Volume94
Issue number1
StatePublished - Jul 1 1999
Externally publishedYes

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ASJC Scopus subject areas

  • Hematology

Cite this

Kohn, D. B., Bauer, G., Rice, C. R., Rothschild, J. C., Carbonaro, D. A., Valdez, P., Hao, Q. L., Zhou, C., Bahner, I., Kearns, K., Brody, K., Fox, S., Haden, E., Wilson, K., Salata, C., Dolan, C., Wetter, C., Aguilar-Cordova, E., & Church, J. (1999). A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34+ cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood, 94(1), 368-371.