Mice Regain Sight After Nanoparticle Gene Therapy

Scientists have developed a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa, an inherited disease characterized by progressive vision loss and eventual blindness, for which there is no cure. The researchers say the findings are based on "a clinically relevant treatment paradigm" and may one day lead to a gene replacement therapy for human retinal degeneration. The research is detailed in The FASEB Journal. Mice with with the retinal degeneration slow (Rds) gene, which causes retinitis pigmentosa, received one of three types of "treatments:" nanoparticles containing the normal copy of the Rds gene, the normal gene alone, or saline solution. read more

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