Gene therapy for retinal disease

Transl Res. 2013 Apr;161(4):241-54. doi: 10.1016/j.trsl.2012.12.007. Epub 2013 Jan 8.


Gene therapy strategies for the treatment of inherited retinal diseases have made major advances in recent years. This review focuses on adeno-associated viral (AAV) vector approaches to treat retinal degeneration and, thus, prevent or delay the onset of blindness. Data from human clinical trials of gene therapy for retinal disease show encouraging signs of safety and efficacy from AAV vectors. Recent progress in enhancing cell-specific targeting and transduction efficiency of the various retinal layers plus the use of AAV-delivered growth factors to augment the therapeutic effect and limit cell death suggest even greater success in future human trials is possible.

Publication types

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

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Genetic Therapy*
  • Genetic Vectors / genetics
  • Humans
  • Neuroprotective Agents / pharmacology
  • Neuroprotective Agents / therapeutic use
  • Promoter Regions, Genetic / genetics
  • Retinal Diseases / drug therapy
  • Retinal Diseases / genetics*
  • Retinal Diseases / therapy*


  • Neuroprotective Agents