In a finding that could lead to treatment of genetic blindness, American researchers have developed a gene therapy for an inherited retinal disease that leads to eventual blindness.
In a research, published in the FASEB Journal, the scientists at the University of Oklahoma Health Sciences Center used a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa.
“We hope the results of our study will be instrumental in generating a cure for the debilitating blindness associated with retinitis pigmentosa and other inherited and acquired retinal diseases,” said senior author Muna I Naash.
“Compacted DNA nanoparticles are an exciting treatment strategy for these diseases and we look forward to exciting new developments,” Naash said.
In the study, the team treated a group of mice with the retinal degeneration slow (Rds) gene, which causes retinitis pigmentosa. The mice received one of three types of treatments — nanoparticles containing the normal copy of the Rds gene, the normal gene alone, or saline solution.
After these treatments were delivered to the mice, the structure and function of the retina were analysed by comparing them to untreated mice with retinitis pigmentosa and healthy mice with the normal Rds gene.
They also measured the level and pattern of Rds gene expression, as well as functional, structural and biochemical improvements in disease symptoms and found that the mice receiving the nanoparticle gene therapy showed significant signs of healing.
These mice had structural improvement in their retinas, as well as functional vision improvements, which lasted throughout the duration of the study.
The mice that received the gene alone or saline continued to lose their vision. The nanoparticles were safe and well-tolerated with no adverse effects.
According to the National Institutes of Health Office of Rare Diseases Research, retinitis pigmentosa is a group of inherited eye diseases that affect the retina.
Retinitis pigmentosa causes cells in the retina to die prematurely, eventually leading to vision loss. There is no cure for it till now. Zeenews
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