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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.
This technology is poised to advance RPGR or RP2 gene therapy to clinical stage using AAV8 or AAV9 vector carrying human full-length RPGR or RP2-coding sequence. The investigators have performed a wide dose range study over 18-months and found it to preserve rod and/or cone function as evidenced by ERG and/or OCT, optomotor tests. Morphologically, the treatment preserved rod and cone viability, and corrected mistrafficking of cone opsin and/or rhodopsin. The therapeutic effect was also achieved in advanced disease stage. The broad treatment window and long-lasting therapeutic effects make the RPGR and RP2 gene therapy attractive for clinical development.
This technology is available for licensing, or the NEI research team will entertain potential collaborations that will advance this technology through the remaining preclinical stage toward IND development under a CRADA or a license combined with a CRADA project.
Pre-clinical (in vivo)
Li L, et al. Ablation of the X-linked retinitis pigmentosa 2 (Rp2) gene in mice results in opsin mislocalization and photoreceptor degeneration
Zhang H, et al. Mistrafficking of prenylated proteins causes retinitis pigmentosa 2.
Mookherjee S, et al. Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy
Wu Z, et al. A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration
Licensing and research collaboration
Original Article: RP2 and RPGR Vectors For Treating X-linked Retinitis PigmentosaNEXT ARTICLE
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