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Newly Improved Method and Composition for Treating Genetically Linked Diseases of the Eye

To improve the transduction efficiency the inventors at the National Eye Institute (NEI) have developed a novel, non-invasive approach of applying electric current in combination with a gene therapy vector. This minimally invasive strategy significantly improves the transduction efficiency of AAV vectors in the mouse retina. This represents an improved method for restoring high levels of RS1 expression in the retina of X-linked retinoschisis (XLRS) patients. The NEI seeks a licensing and/or co-development partner to commercialize its AAV-RS1 Gene Therapy for XLRS.