Ophthalmic disease

Mouse studies identified a LIF-based fusion protein that could help treat blindness. The fusion protein consists of human LIF fused to bacterial dihydrofolate reductase (DHFR), which is stabilized by the antibiotic Primsol trimethoprim. In a mouse model of light-induced blindness, intravitreal delivery of the fusion protein encoded in an AAV2 vector plus Primsol increased outer nuclear layer thickness and cone cell numbers in the eye and visual tracking eye movements compared with vehicle. In a mouse model of retinal degeneration, the same treatment increased outer nuclear layer thickness and cone cell numbers in the eye compared with the fusion protein alone. Next steps could include testing the fusion protein in additional animal models of blindness...