In a project aimed at preventing inherited retinal complications through gene therapy, Maryam Haghshenas, the senior Iranian researcher, emphasized the significant growth of gene therapy applications in the treatment of genetic and acquired diseases, particularly in ophthalmology.
Haghshenas highlighted the importance of producing recombinant viral vectors optimized for effective gene transfer as one of the most effective measures in treating the "retinitis pigmentosa" diseases.
She also discussed the use of a virus carrying a healthy version of the RPGR gene to treat X-linked retinitis pigmentosa caused by a defect in the RPGR gene.
Haghshenas explained that this virus can be less invasively injected into the vitreous, expressing the gene specifically in the photoreceptors of the retina. This would help prevent the blindness associated with retinitis pigmentosa.
She noted that 'retinitis pigmentosa' is a common form of inherited blindness, affecting about one in 2,500 births, causing progressive retinal degeneration and ultimately leading to complete blindness.
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