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    gene augmentation therapy tagged posts

    Restoration of Retinal and Visual Function following Gene Therapy

    October 20, 2020, Categories  Biology/Biotechnology, Health/Medical

    Base editing restores vision
    A mutation in an inherited blindness mouse model abolishes the expression of RPE65, a key enzyme in a visual cycle, in the RPE cells. Base editing treatment can correct the mutation and restore functional RPE65 (green), thereby restoring vision in mice

    New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases. A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

    Published today in Nature Biomedical Engineering, the paper, titled, “Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing,” illustrates the use of a new generation CRISPR...

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