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jabfm vol. 10 issue 2, 2012


We still have a long way to go to effectively deliver genes!


We still have a long way to go to effectively deliver genes!

J Appl Biomater Funct Mater 2012; 10(2): 82 - 91

Article Type: REVIEW

DOI:10.5301/JABFM.2012.9707

Authors

Daniele Pezzoli, Roberto Chiesa, Luigi De Nardo, Gabriele Candiani

Abstract

Gene therapy is emerging as a revolutionary alternative to conventional therapeutic approaches. However, its clinical application is still hampered by the lack of safe and effective gene delivery techniques. Among the plethora of diverse approaches used to ferry nucleic acids into target cells, non-viral vectors represent promising and safer alternatives to viruses and physical techniques. Both cationic lipids and polymers spontaneously wrap and shrink the genetic material in complexes named lipoplexes and polyplexes, respectively, thereby protecting it and shielding its negative charges. The development of non-viral vectors commenced more than two decades ago. Since then, some major classes of interesting molecules have been identified and modified to optimize their properties. However, the way towards the final goal of gene delivery, i.e. protein expression or gene silencing, is filled with obstacles and current non-viral carriers still have concerns about their overall efficiency. We strongly believe that the future of non-viral gene delivery relies on the development of multifunctional vectors specifically tailored with diverse functionalities that act more like viruses. Although these vectors are still a long way from clinical practice they are the ideal platform to effectively shuttle the genetic material to target cells in a safe and controlled way.
In this review, after briefly introducing the basis of gene delivery and therapeutic applications we discuss the main polymeric and lipidic vectors utilized for gene delivery, focusing on the strategies adopted to overcome the major weaknesses inherent to their still limited activity, on the way towards ideal multifunctional vectors.

Article History

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Authors

  • Pezzoli, Daniele [PubMed] [Google Scholar]
    INSTM (Italian National Consortium for Materials Science and Technology), Research Unit Milano Politecnico, Milano - Italy
  • Chiesa, Roberto [PubMed] [Google Scholar]
    Department of Chemistry, Materials and Chemical Engineering ‘‘Giulio Natta’’, Politecnico di Milano, Milano - Italy
  • De Nardo, Luigi [PubMed] [Google Scholar]
    INSTM (Italian National Consortium for Materials Science and Technology), Research Unit Milano Politecnico, Milano and Department of Chemistry, Materials and Chemical Engineering ‘‘Giulio Natta’’, Politecnico di Milano, Milano - Italy
  • Candiani, Gabriele [PubMed] [Google Scholar]
    INSTM (Italian National Consortium for Materials Science and Technology), Research Unit Milano Politecnico, Milano - Italy; Department of Chemistry, Materials and Chemical Engineering ‘‘Giulio Natta’’, Politecnico di Milano, Milano - Italy and The Protein Factory, Centro Interuniversitario di Biotecnologie Proteiche, Politecnico di Milano and Università degli Studi dell’Insubria, Varese - Italy

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