Gene therapy targeting peripheral blood CD34+ hematopoietic stem cells of HIV-infected individuals

Hum Gene Ther. 1997 Dec 10;8(18):2229-38. doi: 10.1089/hum.1997.8.18-2229.

Abstract

Gene therapy is a promising treatment modality for acquired immunodeficiency syndrome (AIDS). Autologous transplantation with genetically altered pluripotent hematopoietic stem cells encoding anti-human immunodeficiency virus (HIV) genes could in theory completely and permanently reconstitute all blood lineages and immune functions with cells resistant to HIV. Recent studies showed that CD34+ stem cell can be mobilized in HIV-infected individuals after granulocyte colony-stimulating factor (G-CSF) administration without major side effects or increase of viral load. In this study, peripheral blood CD34+ cells of five HIV-infected individuals were mobilized with G-CSF and after leukapheresis and enrichment, subjected to retroviral transduction with genes encoding anti-HIV ribozyme-decoy fusion molecules. These cells were tested for the ability to give rise to progeny cells, for retroviral transduction efficiency, and for expression of the transgene. CD34+-derived macrophage-like cells were also challenged with HIV. Results showed that CD34+ cells from HIV-infected individuals gave rise to similar numbers of progeny colonies as cells from healthy donors. The transduction efficiency of these cells varied from 68.8 to 100% as assessed by DNA polymerase chain reaction (PCR) of the transgene in individual colonies. CD34+-derived macrophages expressed anti-HIV genes and displayed a substantial and sustained inhibition of HIV replication as compared to untransduced cells. Furthermore, we showed that after thawing, cryopreserved CD34+ cells from these individuals have survival, proliferation, and transduction parameters comparable to fresh cells. Thus, CD34+ cells from HIV-infected patients can be stored for further genetic manipulations with improved vectors or anti-HIV genes as they become available.

Publication types

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

MeSH terms

  • Animals
  • Antigens, CD34*
  • Cell Line
  • Cell Transformation, Viral
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Granulocyte Colony-Stimulating Factor / administration & dosage
  • HIV Infections / blood*
  • HIV Infections / therapy
  • HIV-1 / pathogenicity
  • Hematopoietic Stem Cells / cytology*
  • Humans
  • Leukapheresis
  • Leukocyte Count
  • Leukocytes, Mononuclear / cytology
  • Leukocytes, Mononuclear / metabolism
  • Macrophages / cytology
  • Macrophages / metabolism
  • Mice
  • Moloney murine leukemia virus
  • Monocytes / cytology
  • Monocytes / metabolism
  • RNA, Catalytic / biosynthesis*
  • RNA, Catalytic / genetics
  • Tumor Cells, Cultured

Substances

  • Antigens, CD34
  • RNA, Catalytic
  • Granulocyte Colony-Stimulating Factor