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A Clinical Trial of Retroviral-Mediated Transfer of arev-Responsive Element Decoy Gene Into CD34+Cells From the Bone Marrow of Human Immunodeficiency Virus-1–Infected Children

1999; Elsevier BV; Volume: 94; Issue: 1 Linguagem: Inglês

10.1182/blood.v94.1.368.413a47_368_371

1528-0020

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

Parvovirus B19 Infection Studies

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 arev-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 1 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.