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Oncotarget. 2015 Oct 13;6(31):30675-703. doi: 10.18632/oncotarget.5169.

Progresses towards safe and efficient gene therapy vectors.

Author information

1
Research Center for Functional Genomics, Biomedicine and Translational Medicine, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.
2
Department of Immunology and Institute for Cellular and Molecular Medicine, Faculty of Health Sciences, University of Pretoria, Pretoria, South Africa.
3
Department of Oncology and Pathology, Cancer Center Karolinska, Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden.
4
Department of Molecular Biology and Genetics, Canik Başari University, Samsun, Turkey.
5
BlueBird Bio, Cambridge, MA, USA.
6
Department of Physiopathology, Faculty of Veterinary Medicine, University of Agricultural Science and Veterinary Medicine, Cluj Napoca, Romania.
7
Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
8
Center for RNA Interference and Non-Coding RNAs, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
9
Department of Immunology, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.
10
Department of Functional Genomics and Experimental Pathology, Oncological Institute "Prof. Dr. Ion Chiricuţăƒ", Cluj Napoca, Romania.

Abstract

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.

KEYWORDS:

AAVP; gene therapy; hybrid vectors; non-viral vectors; viral vectors

PMID:
26362400
PMCID:
PMC4741561
DOI:
10.18632/oncotarget.5169
[Indexed for MEDLINE]
Free PMC Article

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