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Design and Potential of Non-Integrating Lentiviral Vectors
Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN 46202, USA
Department of Medicine, Indiana University School of Medicine, Indianapolis, IN 46202, USA
Department of Microbiology and Immunology, Indiana University School of Medicine, Indianapolis, IN 46202, USA
* Author to whom correspondence should be addressed.
Received: 7 November 2013; in revised form: 22 January 2014 / Accepted: 23 January 2014 / Published: 27 January 2014
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.
Keywords: HIV; non-integrating; episome; lentiviral; integrase
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Cite This Article
MDPI and ACS Style
Shaw, A.; Cornetta, K. Design and Potential of Non-Integrating Lentiviral Vectors. Biomedicines 2014, 2, 14-35.
Shaw A, Cornetta K. Design and Potential of Non-Integrating Lentiviral Vectors. Biomedicines. 2014; 2(1):14-35.
Shaw, Aaron; Cornetta, Kenneth. 2014. "Design and Potential of Non-Integrating Lentiviral Vectors." Biomedicines 2, no. 1: 14-35.