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Iranian scientists develop gene therapy to prevent blindness
Haghshenas highlighted the growing significance of gene therapy in treating genetic and acquired diseases, particularly in ophthalmology. She emphasized the importance of producing recombinant viral vectors optimized for effective gene transfer as a crucial step in treating retinitis pigmentosa, IRNA wrote.
The research focused on X-linked retinitis pigmentosa, caused by a defect in the RPGR gene. Haghshenas and her team created a virus carrying a healthy version of the RPGR gene. This virus can be injected into the vitreous, allowing the gene to be expressed specifically in the photoreceptors of the retina, thus preventing blindness.
Retinitis pigmentosa affects about one in every 2,500 births, causing progressive retinal degeneration and eventual complete blindness. The development of optimized viral vectors offers a promising approach to preventing blindness in patients with this inherited condition.
The team’s invention involves creating mutations in the viral coating to enhance penetration and increase the transfer of the virus to different layers of the retina after intravitreal injection. The virus was successfully tested in a pre-clinical study on one-month-old mice, demonstrating its ability to effectively transfer and express the RPGR gene in the photoreceptor cells of the retina.
This innovative approach eliminates the need for highly invasive subretinal injections, reducing the risk of severe inflammation and retinal detachment. The optimized viral vectors offer higher stability and permeability, making them well-suited for direct injection into the vitreous, a less invasive and safer approach.