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  1. Article

    Open Access

    Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing

    Mutations in the gene for Retinitis Pigmentosa GTPase Regulator (RPGR) cause the X-linked form of inherited retinal degeneration, and the majority are frameshift mutations in a highly repetitive, purine-rich regi...

    Jessica D. Gumerson, Amal Alsufyani, Wenhan Yu, **gqi Lei, Xun Sun… in Gene Therapy (2022)

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