Vector systems for prenatal gene therapy: principles of non-viral vector design and production

Methods Mol Biol. 2012:891:133-67. doi: 10.1007/978-1-61779-873-3_7.

Abstract

Gene therapy vectors based on viruses are the most effective gene delivery systems in use today and although efficient at gene transfer their potential toxicity (Hacein-Bey-Abina et al., Science 302:415-419, 2003) provides impetus for the development of safer non-viral alternatives. An ideal vector for human gene therapy should deliver sustainable therapeutic levels of gene expression without affecting the viability of the host at either the cellular or somatic level. Vectors, which comprise entirely human elements, may provide the most suitable method of achieving this. Non-viral vectors are attractive alternatives to viral gene delivery systems because of their low toxicity, relatively easy production, and great versatility. The development of more efficient, economically prepared, and safer gene delivery vectors is a crucial prerequisite for their successful clinical application and remains a primary strategic task of gene therapy research.

MeSH terms

  • Animals
  • Chitosan / administration & dosage
  • DNA Nucleotidyltransferases / metabolism
  • DNA, Circular / administration & dosage
  • DNA, Circular / biosynthesis
  • DNA, Circular / genetics
  • Endotoxins / isolation & purification
  • Fetus / metabolism
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors / biosynthesis*
  • Genetic Vectors / genetics*
  • Humans
  • Injections
  • Mice
  • Organ Specificity
  • Plasmids / genetics
  • Polyethyleneimine / administration & dosage
  • Prenatal Care / methods*
  • Viruses / metabolism
  • Yolk Sac / blood supply

Substances

  • DNA, Circular
  • Endotoxins
  • Polyethyleneimine
  • Chitosan
  • DNA Nucleotidyltransferases
  • FLP recombinase