Development of a novel trans-lentiviral vector that affords predictable safety

Mol Ther. 2000 Jul;2(1):47-55. doi: 10.1006/mthe.2000.0095.

Abstract

Lentiviral vectors derived from human immunodeficiency virus type 1 (HIV-1) hold great promise for gene therapy. However, the possibility of generating replication-competent retrovirus (RCR) through genetic recombination raises concerns for safety. Here we describe a novel HIV-based packaging system (trans-lentiviral) that splits gag/gag-pol into two parts: One that expresses gag/gag-pro and another that expresses reverse transcriptase and integrase as fusion partners of viral protein R (Vpr). Using a sensitive assay developed to specifically detect recombinant lentiviral DNA mobilization, we demonstrated that the trans-lentiviral vector prevents the generation of recombinants that contain a functional gag-pol structure, while the lentiviral vector generates env-minus recombinant lentivirus that mobilizes recombinant genomes to other cells when pseudotyped with an exogenous envelope. Since an intact gag-pol structure is absolutely required for retroviral DNA mobilization and RCR, the trans-lentiviral vector design significantly reduces this risk. Moreover, it makes it possible to assess the risk of RCR and DNA mobilization using an in vitro assay that monitors trans-lentiviral vector stocks for the regeneration of the gag-pol structure. Therefore, the trans-lentiviral vector design will ensure the greatest predictable level of safety for the clinical application of retroviral vectors, including HIV-based vectors.

Publication types

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

MeSH terms

  • Antigens, CD34 / metabolism
  • Base Sequence
  • Bone Marrow Cells / virology
  • Cell Line
  • Cell Separation
  • Flow Cytometry
  • Fusion Proteins, gag-pol / genetics
  • Gene Products, vpr / genetics
  • Genetic Vectors* / adverse effects
  • Green Fluorescent Proteins
  • HIV-1 / genetics
  • HeLa Cells
  • Humans
  • Lentivirus / genetics*
  • Luminescent Proteins / metabolism
  • Macrophages / virology
  • Models, Biological
  • Molecular Sequence Data
  • Plasmids / metabolism
  • Recombination, Genetic
  • Viral Proteins / genetics
  • vpr Gene Products, Human Immunodeficiency Virus

Substances

  • Antigens, CD34
  • Fusion Proteins, gag-pol
  • Gene Products, vpr
  • Luminescent Proteins
  • Viral Proteins
  • vpr Gene Products, Human Immunodeficiency Virus
  • Green Fluorescent Proteins