Polysaccharide gene transfection agents

Acta Biomater. 2012 Dec;8(12):4224-32. doi: 10.1016/j.actbio.2012.09.022. Epub 2012 Sep 25.

Abstract

Gene delivery is a promising technique that involves in vitro or in vivo introduction of exogenous genes into cells for experimental and therapeutic purposes. Successful gene delivery depends on the development of effective and safe delivery vectors. Two main delivery systems, viral and non-viral gene carriers, are currently deployed for gene therapy. While most current gene therapy clinical trials are based on viral approaches, non-viral gene medicines have also emerged as potentially safe and effective for the treatment of a wide variety of genetic and acquired diseases. Non-viral technologies consist of plasmid-based expression systems containing a gene associated with the synthetic gene delivery vector. Polysaccharides compile a large family of heterogenic sequences of monomers with various applications and several advantages as gene delivery agents. This chapter, compiles the recent progress in polysaccharide based gene delivery, it also provides an overview and recent developments of polysaccharide employed for in vitro and in vivo delivery of therapeutically important nucleotides, e.g. plasmid DNA and small interfering RNA.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Gene Expression
  • Genetic Diseases, Inborn / genetics
  • Genetic Diseases, Inborn / therapy
  • Genetic Therapy / methods*
  • Genetic Vectors / chemistry*
  • Genetic Vectors / genetics
  • Genetic Vectors / therapeutic use
  • Humans
  • Nucleotides / chemistry
  • Nucleotides / therapeutic use
  • Plasmids / chemistry
  • Plasmids / genetics
  • Plasmids / therapeutic use
  • Polysaccharides / chemistry*
  • Polysaccharides / therapeutic use
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / therapeutic use
  • Transfection / methods*

Substances

  • Nucleotides
  • Polysaccharides
  • RNA, Small Interfering