title: Progresses towards safe and efficient gene therapy vectors.
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.
authors.affiliation: Research Center for Functional Genomics, Biomedicine and Translational Medicine, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.
authors.forename: Sergiu
authors.initials: S
authors.lastname: Chira
authors.affiliation: Department of Immunology and Institute for Cellular and Molecular Medicine, Faculty of Health Sciences, University of Pretoria, Pretoria, South Africa.
authors.forename: Carlo S
authors.initials: CS
authors.lastname: Jackson
authors.affiliation: Department of Oncology and Pathology, Cancer Center Karolinska, Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden.
authors.forename: Iulian
authors.initials: I
authors.lastname: Oprea
authors.affiliation: Department of Molecular Biology and Genetics, Canik Başari University, Samsun, Turkey.
authors.forename: Ferhat
authors.initials: F
authors.lastname: Ozturk
authors.affiliation: Department of Immunology and Institute for Cellular and Molecular Medicine, Faculty of Health Sciences, University of Pretoria, Pretoria, South Africa.
authors.forename: Michael S
authors.initials: MS
authors.lastname: Pepper
authors.affiliation: BlueBird Bio, Cambridge, MA, USA.
authors.forename: Iulia
authors.initials: I
authors.lastname: Diaconu
authors.affiliation: Research Center for Functional Genomics, Biomedicine and Translational Medicine, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.
authors.forename: Cornelia
authors.initials: C
authors.lastname: Braicu
authors.affiliation: Research Center for Functional Genomics, Biomedicine and Translational Medicine, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.|Department of Physiopathology, Faculty of Veterinary Medicine, University of Agricultural Science and Veterinary Medicine, Cluj Napoca, Romania.
authors.forename: Lajos-Zsolt
authors.initials: LZ
authors.lastname: Raduly
authors.affiliation: Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.|Center for RNA Interference and Non-Coding RNAs, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: George A
authors.initials: GA
authors.lastname: Calin
authors.affiliation: Research Center for Functional Genomics, Biomedicine and Translational Medicine, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.|Department of Immunology, University of Medicine and Pharmacy "Iuliu Haţieganu", Cluj Napoca, Romania.|Department of Functional Genomics and Experimental Pathology, Oncological Institute "Prof. Dr. Ion Chiricuţă", Cluj Napoca, Romania.|Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Ioana
authors.initials: I
authors.lastname: Berindan-Neagoe
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D010957:Plasmids / Q000235:genetics; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics; D012190:Retroviridae / Q000235:genetics; D014779:Virus Replication / Q000235:genetics
chemical_list: D034741:RNA, Small Interfering
keywords: AAVP; gene therapy; hybrid vectors; non-viral vectors; viral vectors