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The therapeutic efficiency of the U1 approach is dose dependent. Increasing concentrations of the fully adapted U1 (U1-RPGR_mut) result in increased levels of correctly spliced RPGR transcripts in the patient cell line. In contrast, similar treatments of the patient and control fibroblasts with wild type U1 (U1-wt) and mock (lentiviral particles without U1 isoforms) have no detectable influence on RPGR exon 10 splicing. RPGR, retinitis pigmentosa GTPase regulator gene.
