Iranian researchers make headway in preventing blindness through gene therapy

A team of scientists in Iran specializing in gene therapy and preventing blindness in patients with hereditary retinitis pigmentosa (RP) disease that affects retina has succeeded in optimizing recombinant viral vectors that can prevent vision loss. 

Maryam Haghshenas, lead researcher of the team from Mashhad University of Medical Sciences in northeastern Iran, told the information center of the Iranian Vice Presidency for Science, Technology and Knowledge-Based Economy, “RP is a type of retinal degeneration and one of the most common forms of hereditary blindness, with a prevalence of about 1 in 2,500 births.” 

One of the most effective measures for treating the disease is to produce optimized recombinant viral vectors for effective gene transfer by creating a healthy version of the gene, she explained. 

Haghshenas further said after injecting the produced virus into the patient’s eye, the gene is expressed in photoreceptors and prevents the destruction of retinal cells. 

The efficiency of the technique was proven in a preclinical study after the viruses from this invention were injected into the eyes of one-month-old mice. 

The achievement is the first in Iran and even improves the shortcomings of gene therapy technologies being practiced in the world.