Research shows genetically engineered human cells can repair myelin in mice

Wednesday 9 October 2024

Caitlin Astbury

Scientists at our Edinburgh Centre for MS Research have used a gene-editing technique to boost myelin repair.

Myelin is the protective coating around nerves. In MS myelin is damaged, making it harder for messages from the brain to get through. Our bodies can repair myelin. But in MS, and as we age, this becomes less effective. And there are currently no treatments that can boost this process.  

What did the researchers do in this study?  

In this study, researchers grew human oligodendrocyte precursor cells (OPCs) in the lab. These are a type of cell found in the brain that normally transform into myelin-making cells called oligodendrocytes. But signals in the environment of MS lesions stop this from happening. 

Professor Anna Williams and her team used a technique called CRISPR to edit a small section of the DNA of these OPCs to make them ignore anti-repair signals. And found that when these cells were transplanted into mouse brains, they were able to improve myelin repair.  

Professor Williams told us: 

"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 modify 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.
Professor Anna Williams, co-lead of the MS Society Edinburgh Centre for MS Research

What does this mean for people with MS?

Dr Laura Wagstaff, a postdoctoral researcher at the University of Edinburgh who worked on the study, says: 

“Our work is a proof of concept. 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.” 

Caitlin Astbury, our Research Communications Manager, says: 

“Current treatments for MS work by targeting the immune system, making it less likely to attack the protective myelin coating around nerves. But we desperately need to find ways to repair the damage to myelin that has already been done.  

“We’re really proud to have funded this innovative study and the results are invaluable in helping us understand how myelin repair could work. 

"More than 150,000 people live with MS in the UK and research like this brings us one step closer to finding treatments that can stop disability progression for everyone.”