Washington: In a finding that could lead totreatment of genetic blindness, American researchers havedeveloped a gene therapy for an inherited retinal disease thatleads to eventual blindness. In a research, published in the FASEB Journal, thescientists at the University of Oklahoma Health SciencesCenter used a non-viral, synthetic nanoparticle carrier toimprove and save the sight of mice with retinitis pigmentosa.
They also measured the level and pattern of Rds geneexpression, as well as functional, structural and biochemicalimprovements in disease symptoms and found that the micereceiving the nanoparticle gene therapy showed significantsigns of healing. These mice had structural improvement in their retinas,as well as functional vision improvements, which lastedthroughout the duration of the study. The mice that received the gene alone or salinecontinued to lose their vision. The nanoparticles were safeand well-tolerated with no adverse effects. According to the National Institutes of Health Officeof Rare Diseases Research, retinitis pigmentosa is a group ofinherited eye diseases that affect the retina. Retinitis pigmentosa causes cells in the retina to dieprematurely, eventually leading to vision loss. There is nocure for it till now. PTI
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