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BioMed Research International
Volume 2014 (2014), Article ID 703253, 13 pages
Research Article

Cationic Polyene Phospholipids as DNA Carriers for Ocular Gene Therapy

1Center for Biomedicine and Structural Biology (CBME), Institute for Biotechnology and Bioengineering (IBB), University of Algarve, 8005-139 Faro, Portugal
2Doctoral Program in Biomedical Sciences, Department of Biomedical Sciences and Medicine, University of Algarve, 8005-139 Faro, Portugal
3ProRegeM Doctoral Program, Department of Biomedical Sciences and Medicine, University of Algarve, 8005-139 Faro, Portugal
4Department of Chemistry, Norwegian University of Science and Technology, 7491 Trondheim, Norway
5Premedical Unit, Weill Cornell Medical College in Qatar, P.O. Box 24144, Doha, Qatar
6Department of Biomedical Sciences and Medicine, University of Algarve, 8005-139 Faro, Portugal

Received 28 April 2014; Accepted 18 June 2014; Published 24 July 2014

Academic Editor: Taiyoun Rhim

Copyright © 2014 Susana Machado et al. 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.


Recent success in the treatment of congenital blindness demonstrates the potential of ocular gene therapy as a therapeutic approach. The eye is a good target due to its small size, minimal diffusion of therapeutic agent to the systemic circulation, and low immune and inflammatory responses. Currently, most approaches are based on viral vectors, but efforts continue towards the synthesis and evaluation of new nonviral carriers to improve nucleic acid delivery. Our objective is to evaluate the efficiency of novel cationic retinoic and carotenoic glycol phospholipids, designated C20-18, C20-20, and C30-20, to deliver DNA to human retinal pigmented epithelium (RPE) cells. Liposomes were produced by solvent evaporation of ethanolic mixtures of the polyene compounds and coformulated with 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) or cholesterol (Chol). Addition of DNA to the liposomes formed lipoplexes, which were characterized for binding, size, biocompatibility, and transgene efficiency. Lipoplex formulations of suitable size and biocompatibility were assayed for DNA delivery, both qualitatively and quantitatively, using RPE cells and a GFP-encoding plasmid. The retinoic lipoplex formulation with DOPE revealed a transfection efficiency comparable to the known lipid references 3β-[N-(N′,N′-dimethylaminoethane)-carbamoyl]-cholesterol (DC-Chol) and 1,2-dioleoyl-sn-glycero-3-ethylphosphocholine (EPC) and GeneJuice. The results demonstrate that cationic polyene phospholipids have potential as DNA carriers for ocular gene therapy.