| خلاصه مقاله | Introduction: Gene therapy has showed remarkable potential in terms of treating ophthalmic and retinal diseases. Due to the accessibility of eye for external injections, surgical interventions, its immunity-privileged position which can tolerate the accommodation of a viral vector, and existing rigid blood-ocular barriers preventing risk of contamination. A number of biotechnological approaches have been directed to produce cell/gene therapy vectors. The present review aimed at scrutinizing recent advances in ophthalmic gene transfer to control retinal disorders.
Materials & Methods: A full literature review was performed from 2000 to 2022 with keywords “gene therapy”, “ophthalmic”, “Retina” across different medical data bases. The redundant and cancer related articles were excluded.
Results: In recent years several retinal disease have been subjected to gene therapy including Retinitis pigmentosa (RP), Leber Congenital Amaurosis (LCA), Age related macular degeneration (AMD), choroidemia, and Stargardt's disease. The main genes targeted for replacement or over expression were MERTK, RPE65 gene, VEGF, Rep1, and CNTF. The utilized vectors were adenoviral or adenoassociated viral vectors also encapsulate cell technology as a novel method. The routes of administrations were often subretinal or vitral, and encasulted implant. Also, several gene therapy approaches are practising in clinical trials, and some approved. For gene therapy against RP, Luxturna an adenoviral vector containing RPE65 normal gene is now in market. Additionally, thanks to encapsulated cell technology RPE cells were genetically modified to express CNTF and encapsulated inpolymer with final intraocular implantion to treat AMD or other related disease.
Conclusion: In conclusion, considerable scientific and clinical advances have been progressed in cell/gene therapy of eye disease. Additionally, several areas of investigation which could improve the success of cell/gene therapy are necessary. |