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Methods and Compositions for Treating Genetically Linked Diseases of the Eye
Abstract: X-linked retinoschisis (XLRS) is an inherited, monogenetic ocular disease caused by mutations in the retinoschisin (RS1) gene, resulting in the development of cystic cavities throughout the retina and leading to juvenile macular degeneration. Approximately 1:15,000 males in the US are affected, classifying the condition as an orphan indication.  The...
Published: 4/8/2024   |   Inventor(s): Paul Sieving, Ronald Bush, Yong Zeng, Peter Colosi
Keywords(s): Clinical, EYE, GENE THERAPY, ophthalmological, Orphan Indication, Sieving, XLRS
Category(s): TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat, Collaboration Sought > Licensing, Application > Therapeutics