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Viruses 2014, 6(12), 4811-4838; doi:10.3390/v6124811

Alpharetroviral Vectors: From a Cancer-Causing Agent to a Useful Tool for Human Gene Therapy

1
Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany
2
Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, 300 Longwood Avenue, Boston, MA 02115, USA
*
Author to whom correspondence should be addressed.
Received: 26 September 2014 / Revised: 7 November 2014 / Accepted: 20 November 2014 / Published: 5 December 2014
(This article belongs to the Special Issue Retroviral Vectors)
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Abstract

Gene therapy using integrating retroviral vectors has proven its effectiveness in several clinical trials for the treatment of inherited diseases and cancer. However, vector-mediated adverse events related to insertional mutagenesis were also observed, emphasizing the need for safer therapeutic vectors. Paradoxically, alpharetroviruses, originally discovered as cancer-causing agents, have a more random and potentially safer integration pattern compared to gammaretro- and lentiviruses. In this review, we provide a short overview of the history of alpharetroviruses and explain how they can be converted into state-of-the-art gene delivery tools with improved safety features. We discuss development of alpharetroviral vectors in compliance with regulatory requirements for clinical translation, and provide an outlook on possible future gene therapy applications. Taken together, this review is a broad overview of alpharetroviral vectors spanning the bridge from their parental virus discovery to their potential applicability in clinical settings. View Full-Text
Keywords: retrovirology; alpharetroviral vector; gene therapy; clinical translation; regulatory requirements; vector safety retrovirology; alpharetroviral vector; gene therapy; clinical translation; regulatory requirements; vector safety
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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MDPI and ACS Style

Suerth, J.D.; Labenski, V.; Schambach, A. Alpharetroviral Vectors: From a Cancer-Causing Agent to a Useful Tool for Human Gene Therapy. Viruses 2014, 6, 4811-4838.

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