Genome editing for Becker muscular dystrophy

Becker muscular dystrophy (BMD) is caused by changes in a gene called dystrophin. Research has shown that some genetic changes in dystrophin can lead to changes in its shape. There is a link between the differences in the shape of dystrophin and the severity of BMD. People with genetic changes, which lead to significant changes in dystrophin shape, experience early onset and faster progressing symptoms than others.

There have been no research breakthroughs in treating the cause of BMD. The research has mainly focused on treating symptoms of BMD. Developing therapies that target the dystrophin DNA, removing the area that contains the genetic change, and creating dystrophin that causes a milder version of the condition is therefore very important.

The aim of the project is to remove a part of dystrophin to reduce the severity of BMD. The work on the project will involve designing molecular scissors using computer tools and testing how they would work in muscle cells ‒ read more on genome editing. Testing how effective the molecular scissors are in BMD muscle cells in changing the shape of the misshaped dystrophin and testing these molecular scissors in animal models of a specific BMD genetic change.

This approach could have a big impact on the development of treatments for BMD in the future. It would be the first therapy of its kind, which works to remove a part of dystrophin to reduce the severity of the condition. Similar approaches have been used to edit the dystrophin gene for Duchenne muscular dystrophy. This approach could also be adjusted so it could be used to treat other genetic conditions.