Update on ocular gene therapy and advances in treatment of inherited retinal diseases and exudative macular degeneration

Curr Opin Ophthalmol. 2016 May;27(3):268-73. doi: 10.1097/ICU.0000000000000256.


Purpose of review: The purpose of this article is to provide an update on ocular gene therapy and discuss current active clinical trials.

Recent findings: The main target for ocular gene therapy involves the retinal pigment epithelium or photoreceptors. The most common method to deliver viral vectors to these cells includes intravitreal injection, subretinal injection, or access from the suprachoroidal space. Recombinant adeno-associated virus and lentivirus can be engineered to maximize gene delivery to specific targets. There are several clinical trials currently aimed at treating inherited and retinal diseases with gene therapy via viral vectors.

Summary: Recent advances in gene therapy have allowed for a better understanding of inherited and proliferative retinal diseases. New techniques have been developed to improve delivery of viral vectors to their cellular targets. There are currently multiple active clinical trials involving gene therapy underway with promising preliminary results.

Publication types

  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Genetic Therapy* / methods
  • Genetic Vectors
  • Humans
  • Macular Degeneration / genetics
  • Macular Degeneration / therapy*
  • Retinal Diseases / genetics
  • Retinal Diseases / therapy*