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
作者： Donald B. Kohn , Gerhard Bauer , C. Robert Rice , J.C. Rothschild , Denise A. Carbonaro
DOI: 10.1182/BLOOD.V94.1.368.413A47_368_371
关键词: Genetic transfer 、 Stem cell 、 CD34 、 Viral vector 、 Transplantation 、 Biology 、 Haematopoiesis 、 Genetic enhancement 、 Bone marrow 、 Immunology
摘要: Genetic modification of hematopoietic stem cells with genes that inhibit replication human immunodeficiency virus-1 (HIV-1) could lead to development T lymphocytes and monocytic resistant HIV-1 infection after transplantation. We performed a clinical trial evaluate the safety feasibility this procedure, using bone marrow from four HIV-1–infected pediatric subjects (ages 8 17 years). obtained marrow, isolated CD34+ cells, in vitro transduction retroviral vector carrying arev-responsive element (RRE) decoy gene, reinfused into these no evidence adverse effects. The levels gene-containing leukocytes peripheral blood samples 1 year gene transfer/cell infusion have been extremely low. These observations support potential performing therapy for but emphasize need improved transfer techniques.
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