Gene-edited cells could halt multiple sclerosis progression

 Scientists have used gene-editing techniques to boost the repair of nerve cells damaged in multiple sclerosis, a study shows. The innovative method, which was tested in mice, supports the development of cells that can repair the protective myelin coating around nerves, restoring their ability to conduct messages to the brain.

The findings, now published in Nature Communications, offer a potential route for future treatments to stop disability progression, experts say.

Our bodies have the ability to repair myelin, but in multiple sclerosis (MS), and as we age, this becomes less effective. There are currently no treatments to boost this process.

The research team looked at human oligodendrocyte progenitor cells (OPCs) which are found in the brain and normally transform into oligodendrocytes, which produce myelin. But anti-repair signals found in MS lesions—areas of inflammation and damage—stop this from happening.

Researchers from the University of Edinburgh used a technique called CRISPR to edit a small section of the DNA of human, lab-grown OPCs to make them ignore anti-repair signals.

When the gene-edited OPCs were transplanted into mouse brains, the team found they improved the repair of myelin around nerves.

Current treatments for MS work by targeting the immune system, making it less likely to attack the protective myelin coating. This research is a step closer to finding a treatment that can repair the damage to myelin that has already been done, experts say.

"Many studies in the past have tried to transplant oligodendrocytes or similar cells into the brain to repair myelin. However, the hostile environment of MS lesions stops these transplanted cells from working.

"The difference in our study—which was six years in the making—is that we were able to genetically edit the transplanted cells so that they would ignore these negative signals and repair myelin. 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," says Professor Anna Williams.

"Our work is a proof of concept, and the next step is to see if we can remove the need for transplants and edit the cells directly in humans. This is an approach similar to gene therapy which may be an effective method of promoting remyelination in the future," says Dr. Laura Wagstaff.

More information: Laura J. Wagstaff et al, CRISPR-edited human ES-derived oligodendrocyte progenitor cells improve remyelination in rodents, Nature Communications (2024). DOI: 10.1038/s41467-024-52444-w

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