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Induced Pluripotent Stem Cells Derived from Patients with CEP290-associated Ciliopathies and Unaffected Family Members

Abstract: Approximately one-third of non-syndromic retinal dystrophies involve a defect in a ciliary protein. Non-syndromic retinal ciliopathies include retinitis pigmentosa, cone dystrophy, cone-rod dystrophy, macular dystrophy, and Leber-congenital amaurosis (LCA). Many CEP290-LCA patients also exhibit auditory and olfactory defects. Induced pluripotent...

Published: 4/8/2024 | Inventor(s): Anand Swaroop, Hiroko Shimada-Ishii, Yu Holly Chen, Milton English

Keywords(s): Anosmia, Cell therapy, Ciliopathies, Congenital Blindness, Drug Development, GENE THERAPY, hearing loss, iPS, National Eye Institute, NEI, Pluripotent Stem Cells, Primary Cilia, Retina, Retinal degeneration, Swaroop, VISION

Category(s): Application > Research Materials, Collaboration Sought > Licensing, Collaboration Sought > Collaboration, TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat

RP2 and RPGR Vectors For Treating X-linked Retinitis Pigmentosa

Abstract: 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...

Published: 4/8/2024 | Inventor(s): Zhijian Wu, Anand Swaroop, Suddhasil Mookherjee, Suja Hiriyanna, Tiansen Li

Keywords(s): GENE THERAPY, hereditary ocular disease, Pigmentosa, Retina, Wu

Category(s): TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat, Collaboration Sought > Licensing, Collaboration Sought > Collaboration, Application > Therapeutics