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    Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa

    The X-linked RP3 gene codes for the ciliary protein RPGR and accounts for over 10% of inherited retinal degenerations. The critical RPGR-ORF15 splice variant contains a highly repetitive purine-rich linker region...

    B S Pawlyk, O V Bulgakov, X Sun, M Adamian, X Shu, A J Smith, E L Berson… in Gene Therapy (2016)