Potential therapeutic application of antisense oligonucleotides in the treatment of ocular diseases

Expert Opin Pharmacother. 2001 Feb;2(2):277-91. doi: 10.1517/14656566.2.2.277.


Antisense oligonucleotides are a class of compounds being developed as therapeutic agents for many types of diseases. Although still relatively early in the clinical characterisation, the power of this technology lies in the ability to utilise genetic information and the known molecular mechanisms of disease to foster efficient and rational drug design. Consideration of novel approaches to treating ocular diseases is of interest because there are many ocular diseases with no satisfactory treatments. The recent availability of animal models of many ocular diseases provides the opportunity to use antisense oligonucleotides to understand the mechanisms of disease pathology and to potentially intervene therapeutically in ocular disease. There are already a number of examples where antisense oligonucleotides have been applied to the study of ocular physiology and disease and there is an antisense oligonucleotide approved for the treatment of cytomegalovirus (CMV) retinitis. We summarise current research in this area and highlight the properties of these compounds that are favourable for use as ocular therapeutics.

Publication types

  • Review

MeSH terms

  • Animals
  • Eye Diseases / drug therapy*
  • Eye Diseases / genetics
  • Eye Diseases / metabolism
  • Gene Expression / drug effects*
  • Humans
  • Oligonucleotides, Antisense / adverse effects
  • Oligonucleotides, Antisense / pharmacokinetics
  • Oligonucleotides, Antisense / therapeutic use*
  • Tissue Distribution


  • Oligonucleotides, Antisense