Design and potential of non-integrating lentiviral vectors

Aaron Shaw, Kenneth Cornetta

Research output: Contribution to journalReview article

19 Scopus citations

Abstract

Lentiviral vectors have demonstrated promising results in clinical trials that target cells of the hematopoietic system. For these applications, they are the vectors of choice since they provide stable integration into cells that will undergo extensive expansion in vivo. Unfortunately, integration can have unintended consequences including dysregulated cell growth. Therefore, lentiviral vectors that do not integrate are predicted to have a safer profile compared to integrating vectors and should be considered for applications where transient expression is required or for sustained episomal expression such as in quiescent cells. In this review, the system for generating lentiviral vectors will be described and used to illustrate how alterations in the viral integrase or vector Long Terminal Repeats have been used to generate vectors that lack the ability to integrate. In addition to their safety advantages, these non-integrating lentiviral vectors can be used when persistent expression would have adverse consequences. Vectors are currently in development for use in vaccinations, cancer therapy, site-directed gene insertions, gene disruption strategies, and cell reprogramming. Preclinical work will be described that illustrates the potential of this unique vector system in human gene therapy.

Original languageEnglish (US)
Pages (from-to)14-35
Number of pages22
JournalBiomedicines
Volume2
Issue number1
DOIs
StatePublished - Mar 1 2014

Keywords

  • Episome
  • HIV
  • Integrase
  • Lentiviral
  • Non-integrating

ASJC Scopus subject areas

  • Medicine (miscellaneous)
  • Biochemistry, Genetics and Molecular Biology(all)

Fingerprint Dive into the research topics of 'Design and potential of non-integrating lentiviral vectors'. Together they form a unique fingerprint.

  • Cite this