Developing next-generation therapies for LMNA-related congenital muscular dystrophy using ‘mini muscle’ models

To find effective treatments, researchers need to test treatments and gather evidence that they could work in people. Models can be used to mimic the condition. Normally animal models are used, however there are important difference between humans and animals. What works in a mouse, might not work in a human. Professor Tedesco has previously shown that ‘mini muscles’ can be grown in a dish from skin cells of people with LMNA-related congenital muscular dystrophy. These mini muscles can more accurately mirror what happens in people with the condition, which should help to identify the most promising treatments.

This project aims to further develop the mini muscles grown in a dish so that they look more closely like actual muscles. Professor Tedesco and team will also focus on making sure the mini muscles mirror the features of LMNA-related congenital muscular dystrophy. This is key to creating a model which can help to determine if a potential treatment is having an effect.

Once they have improved the mini muscles, they will use the model to test how treatments can be delivered to the muscles. The team will then test several different potential treatments to see if they work, paying particular attention to safety.

The development of potential treatments is expensive and lengthy, and often treatments that make it to clinical trials don’t perform as they did in models. This research aims to improve a new model that mirrors what happens in people with LMNA-related congenital muscular dystrophy. The researchers think using the model will improve their chances of finding treatments that work for people with the condition, when tested later in clinical trials.

This project is funded in collaboration with LifeArc.