Transplantation of "super-OPCs" to improve central nervous system remyelination.

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Multiple sclerosis (MS) is a common chronic disease of the brain and spinal cord, often starting in young adulthood, which frequently causes disability. Although the early stages of the disease can now be controlled with medication, in the later stages, when patients gradually develop more disability over time, there are no treatments to either slow, stop or reverse disability. One focus of research to help the later "progressive' phase of MS is to encourage repair of the myelin sheath that covers the nerves, similar to the insulation on electrical wires. This is damaged in MS and when replaced can protect nerves from dying, and avoid disability. The cells that carry out repair of this myelin sheath are called oligodendrocytes, which come from oligodendrocyte progenitor cells (OPCs), a form of stem cell. These cells are present in brains, but they struggle to repair the myelin sheaths in MS as they are cannot function well in the toxic environment in MS damaged areas.In this project, we will test whether we can genetically change OPCs in a dish to make them unable to be harmed by a toxic MS environment. We will then transplant them into the brain and see whether this makes them more able to repair. We aim to do this by genetically modifying them to both help them to reach the areas of damage and then to make more myelin when they arrive. We will do this using human cells, testing them in a mouse model of MS, but if this is successful, we will then aim to do try this in a trial in humans.This project will find out whether transplants of genetically modified OPCs into brains might in the future be turned into a helpful therapy for progressive MS.