Delivery of neurotrophic factors and therapeutic proteins for retinal diseases

Expert Opin Biol Ther. 2005 Nov;5(11):1443-52. doi: 10.1517/14712598.5.11.1443.


Neurotrophic factors have the ability to protect and initiate growth of neurons. In the central nervous system, neurotrophic factors are neuroprotective in a wide range of disease states. Similarly, disease pathologies of the neurosensory retina respond favourably in animal models of retinitis pigmentosa, macular degeneration, glaucoma and others. With advances in drug delivery and cell therapy, an almost universal increase in efficacy is being realised. Now, repeated injections of neurotrophic factors are being replaced by controlled delivery of cell-mediated factor secretion, reducing the number of potential acute side effects. Tissue engineering strategies in conjunction with gene-modulated protein therapy or gene transfer are creating a unique treatment niche and are quickly gaining acclaim in the clinic. This review surveys the founding and current work on neurotrophic factors such as CNTF, BDNF, GDNF, LEDGF, PEDF and others. Ongoing clinical trials and successful preclinical studies are summarised as well.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Delivery Systems / methods*
  • Drug Delivery Systems / trends
  • Genetic Therapy / methods
  • Humans
  • Nerve Growth Factors / administration & dosage*
  • Nerve Growth Factors / genetics
  • Protein Engineering / methods*
  • Protein Engineering / trends
  • Retinal Diseases / drug therapy*
  • Retinal Diseases / genetics


  • Nerve Growth Factors