The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of a targeted disruption of the Rpe65 gene in mice, a novel model for studying retinal diseases and testing potential therapies.

This technology includes the targeted disruption of the Rpe65 gene in mice, which serves as a crucial model for studying inherited retinal diseases and testing potential therapies. The RPE65 protein is essential for converting dietary vitamin A into the 11-cis isomer, a vital component for vision. The absence of this protein in our mouse model leads to a complete lack of 11-cis retinoids, resulting in blindness, thus confirming its role in the visual cycle. This innovative genetic strategy not only provides insights into the biochemical processes of vision but also opens avenues for developing treatments for RPE65-related retinal disorders. 

The technical solution involves creating a genetically modified mouse model that lacks the Rpe65 gene, allowing researchers to observe the resulting phenotype and understand the implications of RPE65 mutations in human diseases. This model is particularly valuable for pharmaceutical companies and research institutions focused on ocular health, as it enables the testing of new drugs and therapies aimed at restoring vision in patients with RPE65 mutations. By utilizing this model, stakeholders can accelerate the development of effective treatments for retinal diseases, ultimately benefiting patients suffering from vision loss. 

We are seeking licensing opportunities for this groundbreaking technology, which has already garnered interest from companies such as Acucela Inc. Collaborators can expect a mutually beneficial partnership that leverages our expertise in genetic engineering and retinal biology to advance therapeutic solutions. Licensing this technology will provide access to a unique research tool that can significantly impact the field of ophthalmology and vision science.