Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

Nat Biotechnol. 1997 Sep;15(9):871-5. doi: 10.1038/nbt0997-871.


Retroviral vectors derived from lentiviruses such as HIV-1 are promising tools for human gene therapy because they mediate the in vivo delivery and long-term expression of transgenes in nondividing tissues. We describe an HIV vector system in which the virulence genes env, vif, vpr, vpu, and nef have been deleted. This multiply attenuated vector conserved the ability to transduce growth-arrested cells and monocyte-derived macrophages in culture, and could efficiently deliver genes in vivo into adult neurons. These data demonstrate the potential of lentiviral vectors in human gene therapy.

Publication types

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

MeSH terms

  • Animals
  • Cells, Cultured
  • Corpus Striatum / drug effects
  • Corpus Striatum / metabolism
  • DNA, Viral / genetics
  • Gene Deletion
  • Gene Expression Regulation, Viral / drug effects
  • Gene Expression Regulation, Viral / genetics*
  • Genes, Reporter / genetics
  • Genetic Therapy*
  • Genetic Vectors / genetics*
  • Genetic Vectors / pharmacology
  • HIV-1 / genetics*
  • Humans
  • Macrophages
  • Rats
  • Transduction, Genetic
  • Transfection
  • Viral Envelope Proteins / genetics


  • DNA, Viral
  • Viral Envelope Proteins