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Treating MS with gene-edited brain cells

Brain scan

UK researchers have used gene editing to alter human brain cells and successfully repair damaged nerves in mice – a potential treatment for multiple sclerosis (MS).


MS affects the brain and the spinal cord, and involves the body’s immune system attacking the protective coating on nerve cells, called myelin. This leads to various symptoms, from mild to severe, including problems with sight, speech or movement.


The study, at the University of Edinburgh, and also involving the University of Cambridge, looked to a type of cell in the brain called human oligodendrocyte progenitor cells (OPCs), which usually turn into myelin-producing oligodendrocyte cells. However, the presence of ‘anti-repair’ signals in MS prevent these new myelin from being formed.


The gene-edited OPCs were able to ignore these ‘anti-repair’ signals and were transplanted by the researchers into the brains of mice. The team then saw that there was improved repair of myelin.


This points to a MS treatment that can repair myelin, when the damage has already been done, in contrast to current treatments that try to control the attack by the immune system.


Anna Williams, at Edinburgh, said: “This is exciting as now we have shown that we can scientifically tweak cells in a dish and transplant them into models to improve repair.”

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