Delivery Systems for In Vivo Use of Nucleic Acid Drugs

  • Resende R.R Departamento de Fisiologia e Biofísica, Instituto de Ciências Biomédicas, Universidade de São Paulo, 05508-900 São Paulo, SP, Brazil.
  • Torres H.A.M Departamento de Biofísica, Universidade Federal de São Paulo, São Paulo 04023-062, SP, Brazil.
  • Yuahasi K.K Departamento de Neurologia e Neurocirurgia, Universidade Federal de São Paulo, São Paulo, SP, Brazil.
  • Majumder P Departamento de Bioquímica, Instituto de Química, Universidade de São Paulo, São Paulo 05508-900, SP, Brazil.
  • Ulrich H Departamento de Bioquímica, Instituto de Química, Universidade de São Paulo, São Paulo 05508-900, SP, Brazil.
Keywords: aptamers, RNA interference (RNAi), drug delivery systems, nucleic-acid-based drugs

Abstract

The notorious biotechnological advance of the last few decades has allowed the development of experimental methods for understanding molecular mechanisms of genes and new therapeutic approaches. Gene therapy is maturing into a viable, practical method with the potential to cure a variety of human illnesses. Some nucleic-acid-based drugs are now available for controlling the progression of genetic diseases by inhibiting gene expression or the activity of their gene products. New therapeutic strategies employ a wide range of molecular tools such as bacterial plasmids containing transgenic inserts, RNA interference and aptamers. A nucleic-acid based constitution confers a lower immunogenic potential and as result of the high stringency selection of large molecular variety, these drugs have high affinity and selectivity for their targets. However, nucleic acids have poor biostability thus requiring chemical modifications and delivery systems to maintain their activity and ease their cellular internalization. This review discusses some of the mechanisms of action and the application of therapies based on nucleic acids such as aptamers and RNA interference as well as platforms for cellular uptake and intracellular delivery of therapeutic oligonucleotides and their trade-offs.
Published
2007-01-01
How to Cite
R.RR., H.A.MT., K.KY., PM., & HU. (2007). Delivery Systems for In Vivo Use of Nucleic Acid Drugs. Drug Target Insights, 2(1). https://doi.org/10.33393/dti.2007.1319