What can SV40-derived vectors do for gene therapy?

David S. Strayer, Mark A. Zern, Jayanta Roy-Chowdhury

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

The limited success of gene therapy as an approach to treating human disease largely reflects the limitations of the gene delivery vectors that have been used. Poor titers, low transduction efficiency, waning transgene expression and immunogenicity have remained obstacles in the field. As a consequence, much research in normal, immunocompetent animals has not demonstrated therapeutic levels of gene delivery, and results from most human clinical trials have been predictably discouraging. Recombinant gene transfer vectors derived from SV40 virus (rSV40) are potentially of great interest for those working in gene therapy, since these vectors are not subject to many of the problems that have limited gene delivery using other vector systems. rSV40 is made at a very high titer and infects - and so transduces - almost all nucleated cell types very efficiently, regardless of lineage or whether they are resting or dividing; they integrate and are not susceptible to transgene silencing, and they elicit no detectable immune response on the part of normal animals and so can be used to deliver multiple transgenes over time and in sequence. The recent development of 'gutless' rSV40 vectors has expanded the range of potential therapeutic transgenes that can be delivered with this system and added flexibility to the expression configurations that can be accommodated. All of these functional characteristics of SV40-derived vectors have their bases in the biology of SV40 and similar viruses, and have important implications for the potential utility of rSV40 vectors in gene therapeutics. Like all viral gene delivery systems, these vectors have their idiosyncrasies and limitations. They also allow gene delivery that bypasses many of the difficulties that have plagued the field from its inception.

Original languageEnglish (US)
Pages (from-to)313-323
Number of pages11
JournalCurrent Opinion in Molecular Therapeutics
Volume4
Issue number4
StatePublished - Aug 2002

Fingerprint

Genetic Therapy
Transgenes
Genes
Simian virus 40
Gene Transfer Techniques
Viral Genes
Therapeutics
Clinical Trials
Research

Keywords

  • Antigenicity
  • Gene delivery
  • Gutless vectors
  • Immunogenicity
  • Simian virus-40

ASJC Scopus subject areas

  • Genetics
  • Genetics(clinical)
  • Pharmacology, Toxicology and Pharmaceutics(all)

Cite this

What can SV40-derived vectors do for gene therapy? / Strayer, David S.; Zern, Mark A.; Roy-Chowdhury, Jayanta.

In: Current Opinion in Molecular Therapeutics, Vol. 4, No. 4, 08.2002, p. 313-323.

Research output: Contribution to journalArticle

Strayer, David S. ; Zern, Mark A. ; Roy-Chowdhury, Jayanta. / What can SV40-derived vectors do for gene therapy?. In: Current Opinion in Molecular Therapeutics. 2002 ; Vol. 4, No. 4. pp. 313-323.
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