Protrudin Gene Therapy Provokes Regrowth in Injured Optic Nerve Cells
Nerves regenerate poorly, and the regrowth of axons linking neurons following injury is hampered by scar formation. Finding a way to force greater regrowth of nerve tissue is an important goal for the regenerative medicine community. A variety of methods have shown some promise in early stage studies, and the example here is one among many. There has to date been comparatively little progress towards the clinic, however.
Glaucoma is a disease caused by progressive damage to the optic nerve, which transfers visual information from the eye to the brain. Conventional treatments focus on reducing eye pressure to prevent optic nerve damage, but they do not work for about 15 per cent of patients and there is currently no way to repair damaged nerve cells.
Researchers tested whether a gene responsible for producing a protein known as protrudin could stimulate the regeneration of nerve cells and stop them from dying when they were injured. They used a cell culture system to grow brain cells in the lab and then injured them using a laser before introducing a gene to increase the amount of protrudin in the cells, vastly increasing their ability to repair and regenerate.
Tests of eye and optic nerve cells found the protein enabled significant regeneration weeks after a crush injury to the optic nerve. The research demonstrated almost complete protection of nerve cells from a mouse retina growing in cell culture, a technique which would usually be expected to result in extensive cell death. Next steps are to explore the ability of protrudin to protect and regenerate human retinal cells.