Abstract
This chapter describes the topic of gene therapy based on colloidal drug delivery, as an alternative to the use of viral carriers. Non-viral vectors are promising transfection agents and do not suffer from limitations related to toxicity and immunogenic effects. In particular, lipid-based aggregates are generally considered biocompatible and versatile nanocarriers whose composition can be designed to include a cationic molecule which ensures strong interaction with nucleic acid. Herein the main issues related to complex formation and in vitro administration are illustrated with key examples, such as liposome-DNA plasmid (pDNA) association and micelles-siRNA complexes.
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Acknowledgments
We would like to thank Laura Ciani (Department of Chemistry University of Florence), Anna Salvati (Research Institute of Pharmacy, University of Groningen), and Angelo Fortunato (Center for Evolution and Cancer, University of California San Francisco) for the optimization of experimental protocols.
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Falsini, S., Ristori, S. (2016). Lipoplexes from Non-viral Cationic Vectors: DOTAP-DOPE Liposomes and Gemini Micelles. In: Candiani, G. (eds) Non-Viral Gene Delivery Vectors. Methods in Molecular Biology, vol 1445. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3718-9_3
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DOI: https://doi.org/10.1007/978-1-4939-3718-9_3
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