Article
Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.
Human molecular genetics - 15 Nov 2015
Mookherjee Suddhasil, Hiriyanna Suja, Kaneshiro Kayleigh, Li Linjing, Li Yichao, Li Wei, Qian Haohua, Li Tiansen, Khanna Hemant, Colosi Peter, Swaroop Anand, Wu Zhijian
Abstract excerpt
Retinal neurodegenerative diseases are especially attractive targets for gene replacement therapy, which appears to be clinically effective for several monogenic diseases. X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor dysfunction primarily caused by mutations in RPGR or RP2 gene. With a goal to develop gene therapy for the XLRP-RP2...
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