Abstract
Ocular neovascularization is a major cause of blindness and visual disability in developed countries. There has been considerable recent progress identifying molecular signals that participate in ocular neovascularization and it appears that imbalances between stimulatory and inhibitory proteins contribute. Re-establishing balance by ocular gene transfer to block stimulators or increase expression of endogenous inhibitors is an appealing therapeutic approach, because it provides a potential means to achieve sustained intraocular effects with little impact on the rest of the body. Proof-of-concept has been provided in animal models using several vector systems and several transgenes and completion of a phase I study testing intraocular injection of an adenoviral vector expressing pigment epithelium-derived factor is an important milestone that will help to accelerate future progress. It is likely that additional vectors and transgenes will enter clinical trials in the near future. This report discusses the rationale and experimental evidence regarding several candidate transgenes.
Keywords: Age-related macular degeneration, angiogenesis, diabetic retinopathy, gene therapy, neovascularization, proliferative retinopathies
Current Gene Therapy
Title: Gene Therapy for Ocular Neovascularization
Volume: 7 Issue: 1
Author(s): Peter A. Campochiaro
Affiliation:
Keywords: Age-related macular degeneration, angiogenesis, diabetic retinopathy, gene therapy, neovascularization, proliferative retinopathies
Abstract: Ocular neovascularization is a major cause of blindness and visual disability in developed countries. There has been considerable recent progress identifying molecular signals that participate in ocular neovascularization and it appears that imbalances between stimulatory and inhibitory proteins contribute. Re-establishing balance by ocular gene transfer to block stimulators or increase expression of endogenous inhibitors is an appealing therapeutic approach, because it provides a potential means to achieve sustained intraocular effects with little impact on the rest of the body. Proof-of-concept has been provided in animal models using several vector systems and several transgenes and completion of a phase I study testing intraocular injection of an adenoviral vector expressing pigment epithelium-derived factor is an important milestone that will help to accelerate future progress. It is likely that additional vectors and transgenes will enter clinical trials in the near future. This report discusses the rationale and experimental evidence regarding several candidate transgenes.
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Campochiaro A. Peter, Gene Therapy for Ocular Neovascularization, Current Gene Therapy 2007; 7 (1) . https://dx.doi.org/10.2174/156652307779940252
DOI https://dx.doi.org/10.2174/156652307779940252 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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