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Gene Therapy Approaches Toward Biomedical Breakthroughs

  • Lies Marguillier
  • Peter Dubruel
  • Sandra Van Vlierberghe
Chapter
Part of the Micro- and Opto-Electronic Materials, Structures, and Systems book series (MOEM)

Abstract

The treatment of diseases at genetic level seems a virtual certainty due to revolutionary advances in cell and molecular biology in the past two decades. Gene therapy has attracted increasing interest as a possible therapy for treating a wide variety of diseases, including both genetic and non-genetic disorders. It is an experimental technique that delivers genetic material to somatic or germ cells of patients, with the intent of altering cellular function or structure at the molecular level to improve a clinical outcome (Anderson, Nature 392, 25–30, 1998). The exogenous genetic material that is introduced into the cells can be either plasmid DNA or one or more specific genes. The latter are often referred to as transgenes. A plasmid, on the other hand, is a DNA molecule which differs from chromosomal DNA and has the ability to replicate independently of it (Sun and Lee, Am. J. Biochem. Biotechnol. 2(2), 66–72, 2006). Several gene therapy approaches have already been evaluated, including the replacement of a mutated gene causing illness with a healthy copy of the gene, the inactivation of an improperly functioning mutated gene, and the introduction of a new gene into the body to help in countering a disease (Abramson et al. Genet. Home Ref. 1(7), 123–131, 2010). Clearly, gene therapy should be applicable in clinical settings and not only in a laboratory. The most important prerequisites include a sufficient DNA delivery and a sustained expression of its secretory products, often proteins. For some purposes, such as the stimulation of vascular growth, short-term activity can suffice, while for others (e.g., the rectification of a genetic defect), long-term gene expression can be required (Strayer, Expert Opin. Investig. Drugs 8(12), 2159–2172, 1999). The current chapter will overview the state-of-the-art covering viral and non-viral gene delivery approaches for a range of biomedical applications.

Keywords

Gene therapy Viral Non-viral Cationic polymers Scaffolds Gene therapy applications 

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  • Lies Marguillier
    • 1
  • Peter Dubruel
    • 2
  • Sandra Van Vlierberghe
    • 2
    • 3
  1. 1.Polymer Chemistry & Biomaterials GroupGhent UniversityGhentBelgium
  2. 2.Polymer Chemistry & Biomaterials Group, Centre of Macromolecular Chemistry, Department of Organic and Macromolecular ChemistryGhent UniversityGhentBelgium
  3. 3.Brussels Photonics, Department of Applied Physics and PhotonicsVrije Universiteit BrusselBrusselBelgium

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