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. 1999 Jul 1;94(1):368-71.


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 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.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Acquired Immunodeficiency Syndrome / genetics
  • Acquired Immunodeficiency Syndrome / immunology
  • Acquired Immunodeficiency Syndrome / pathology
  • Acquired Immunodeficiency Syndrome / therapy*
  • Adolescent
  • Cell Differentiation
  • Child
  • Gene Transfer Techniques
  • Genes, rev*
  • Genetic Therapy*
  • Genetic Vectors
  • HIV Long Terminal Repeat / genetics*
  • HIV-1 / genetics*
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells / pathology
  • Hematopoietic Stem Cells / physiology
  • Humans
  • Pilot Projects
  • Retroviridae
  • T-Lymphocytes / immunology
  • Virus Replication / genetics*