Gene therapy for Becker

A microdystrophin gene therapy approach for Becker muscular dystrophy

We have awarded a grant to the team of Professor Thomas Voit (pictured) at the Great Ormond Street Biomedical Research Centre at UCL to develop a microdystrophin gene therapy for Becker muscular dystrophy. This project will be valuable in helping to understand how crucial gene therapy treatments currently in the pipeline will affect boys and young men with Duchenne muscular dystrophy.

How will this benefit Duchenne patients?

Becker muscular dystrophy is a milder form of muscular dystrophy than Duchenne. This research aims to establish the range of dystrophin expression at which a gene therapy treatment would be beneficial – data that would benefit Duchenne patients as well. In addition, it is thought that the result of gene therapy treatments for Duchenne patients may result in improved dystrophin levels similar to those in Becker patients. A greater understanding of treatments for Becker is therefore vital for tackling future challenges faced by Duchenne patients.

Pre-clinical research

£108,000 contribution

Funded in 2017-18

London, UK

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