Developing a triple-action treatment for Duchenne muscular dystrophy

When the body doesn’t make enough, or any, of a protein called dystrophin, it causes Duchenne muscular dystrophy (DMD). This is because the instructions (gene) for dystrophin are changed. Dystrophin helps protect muscles when they move. Without it, muscles get damaged and stop working properly.

Some treatments can help the body make more dystrophin. These aren’t cures, and they often only make small amounts of the protein. This means they might slow down how quickly the condition gets worse, but they don’t stop it. That’s why scientists are still working hard to find better treatments that can make a bigger difference.

One idea is to create a treatment that does more than one job. It would help the body make more dystrophin and boost other proteins that help muscles stay strong and healthy.

The team aim to create a treatment which does three different jobs:

1. Helps the body make more dystrophin.
The team will use a technique called exon skipping, which helps the body skip over the change in the dystrophin gene so it can still make a shorter, but working, version of the protein. This may help protect muscles and slow down the speed at which they get weaker.

2. Increases the amount of a protein that helps muscles stay strong and resist damage.
The team will use a gene editing tool that attaches to the instructions that make this protein which acts like a turbo switch, helping the body make more of it. They’ll test different turbo switches to find out which ones work best.

3. Increases the amount of a protein that helps muscles grow and reduces scar tissue.
This will also be done using gene editing, with turbo switches designed to increase how much of the protein the body makes. The team will test several versions to see which works best.

Once the treatment is ready, they’ll test it in muscle cells from humans and mice that don’t make dystrophin. They’ll also look at the best way to get the treatment to the muscles, which is important to make sure it works properly.

Treatments for DMD have improved over time. Some can now slow down the speed at which the condition gets worse, but they don’t stop it, and they don’t work for everyone. That’s why scientists are continuing to look for better options.

This project is trying something new. It combines three different ways to help protect muscles. If it works well and future research backs it up, it could lead to a treatment that helps people with DMD stay stronger for longer than current ones can.