Sight of Action: the Rationale and Evolution of Gene Therapy Approaches to the Treatment of Retinal Diseases
| Autor: | Sharmila Vijay, Mark S. Blumenkranz, Kathryn W. Woodburn |
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| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
business.industry Genetic enhancement Transgene Retinal Diabetic retinopathy Macular degeneration medicine.disease Bioinformatics Viral vector Clinical trial 03 medical and health sciences Ophthalmology chemistry.chemical_compound 030104 developmental biology 0302 clinical medicine chemistry 030221 ophthalmology & optometry Medicine Expression cassette business |
| Zdroj: | Current Ophthalmology Reports. 8:267-280 |
| ISSN: | 2167-4868 |
| Popis: | This review will summarize ocular gene therapy clinical trials and associated technology that have been initiated within the last 5 years. Initial programs utilized subretinal administration of an AAV2 serotype coupled with a ubiquitous promoter and a transgene targeting both monogenic inherited retinal diseases and acquired chronic ocular diseases polygenic in origin such as age-related macular degeneration and diabetic retinopathy. Cellular specific viral vectors with optimization of the expression cassette to include cellular-specific promoters and cassette regulatory elements to improve yields and durability with less invasive administration routes are now being clinically evaluated. These developments open the door for potential treatment of rare monogenic diseases and more common polygenic heterogeneous ocular disorders, where in situ expression of therapeutic proteins may reduce the need for repeated intravitreal injections. While initial reports are promising, only time will confirm whether sustained durable “curative” expression is a reality. |
| Databáze: | OpenAIRE |
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