Abstract
The photoreceptor cells of the retina are subject to a wide range of genetic diseases. This review summarizes current knowledge regarding an important group of retinal diseases caused by mutations in photoreceptor-enriched genes. In addition, progress toward treatment of a variety of these diseases in animal models via adeno-associated virus gene therapy is described. Although no human trials have yet been initiated to treat diseases caused by mutations in photoreceptor-enriched genes, there is a great deal of optimism regarding the prospects of treating these diseases using adeno-associated virus gene therapy.
| Original language | English |
|---|---|
| Pages (from-to) | 335-347 |
| Number of pages | 13 |
| Journal | Pharmacogenomics |
| Volume | 9 |
| Issue number | 3 |
| DOIs | |
| State | Published - Mar 2008 |
Keywords
- Adeno-associated virus
- Gene therapy
- Photoreceptors
- Retina
- cis-regulation element