Gene therapy in retinal diseases: A review
| Autor: | Deven Dhurandhar, Niroj Kumar Sahoo, Indumathi Mariappan, Raja Narayanan |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: | |
| Zdroj: | Indian Journal of Ophthalmology, Vol 69, Iss 9, Pp 2257-2265 (2021) |
| Druh dokumentu: | article |
| ISSN: | 0301-4738 1998-3689 |
| DOI: | 10.4103/ijo.IJO_3117_20 |
| Popis: | Over 2 million people worldwide are suffering from gene-related retinal diseases, inherited or acquired, and over 270 genes have been identified which are found to be responsible for these conditions. This review article touches upon the mechanisms of gene therapy, various enzymes of the visual cycle responsible for different genetic diseases, Luxturna—the first US Food and Drug Administration (FDA)-approved therapeutic gene product, and several ongoing trials of gene therapy for age-related macular degeneration. Gene therapy has tremendous potential for retinal conditions due to its ease of accessibility, immune-privileged status, and tight blood-retinal barriers, limiting systemic side effects of the drug. In recent years, advances in gene therapy in retinal conditions have increasing significantly, with progress in cell-specific targeting and transduction efficiency of gene products through the use of adeno-associated viral vectors (AAVs), suggesting that even greater success in future clinical trials is possible. |
| Databáze: | Directory of Open Access Journals |
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