Progresses towards safe and efficient gene therapy vectors

Sergiu Chira, Carlo S. Jackson, Iulian Oprea, Ferhat Ozturk, Michael S. Pepper, Iulia Diaconu, Cornelia Braicu, Lajos-Zsolt Raduly, George A. Calin _ and Ioana Berindan-Neagoe

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Oncotarget. 2015; 6:30675-30703. https://doi.org/10.18632/oncotarget.5169

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Sergiu Chira1, Carlo S. Jackson2, Iulian Oprea3, Ferhat Ozturk4, Michael S. Pepper2, Iulia Diaconu7, Cornelia Braicu1, Lajos-Zsolt Raduly1,10, George A. Calin8,9, Ioana Berindan-Neagoe1,5,6,8

1Research Center for Functional Genomics, Biomedicine and Translational Medicine, University of Medicine and Pharmacy “Iuliu Haţieganu”, Cluj Napoca, Romania

2Department of Immunology and Institute for Cellular and Molecular Medicine, Faculty of Health Sciences, University of Pretoria, Pretoria, South Africa

3Department of Oncology and Pathology, Cancer Center Karolinska, Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden

4Department of Molecular Biology and Genetics, Canik Başari University, Samsun, Turkey

5Department of Immunology, University of Medicine and Pharmacy “Iuliu Haţieganu”, Cluj Napoca, Romania

6Department of Functional Genomics and Experimental Pathology, Oncological Institute “Prof. Dr. Ion Chiricuţă”, Cluj Napoca, Romania

7BlueBird Bio, MA, USA

8Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

9Center for RNA Interference and Non-Coding RNAs, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

10Department of Physiopathology, Faculty of Veterinary Medicine, University of Agricultural Science and Veterinary Medicine, Cluj Napoca, Romania

Correspondence to:

George A. Calin, e-mail: gcalin@mdanderson.org

Sergiu Chira, e-mail: sergiu.chira@umfcluj.ro

Keywords: gene therapy, non-viral vectors, viral vectors, hybrid vectors, AAVP

Received: June 16, 2015     Accepted: August 22, 2015     Published: September 04, 2015


The emergence of genetic engineering at the beginning of the 1970′s opened the era of biomedical technologies, which aims to improve human health using genetic manipulation techniques in a clinical context. Gene therapy represents an innovating and appealing strategy for treatment of human diseases, which utilizes vehicles or vectors for delivering therapeutic genes into the patients' body. However, a few past unsuccessful events that negatively marked the beginning of gene therapy resulted in the need for further studies regarding the design and biology of gene therapy vectors, so that this innovating treatment approach can successfully move from bench to bedside. In this paper, we review the major gene delivery vectors and recent improvements made in their design meant to overcome the issues that commonly arise with the use of gene therapy vectors. At the end of the manuscript, we summarized the main advantages and disadvantages of common gene therapy vectors and we discuss possible future directions for potential therapeutic vectors.

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