Investing in gene therapy to advance treatments for muscle-wasting conditions

 

Update 25/7/17: UNITE-DMD researchers demonstrate efficacy of Duchenne gene therapy

Update 30/6/17: Sarepta partners with Genethon, which strengthens UNITE-DMD’s prospects

We are proud to be a long-standing supporter of research into gene therapy. This technology has advanced significantly over the years and it’s now time to take it to the next level: to test its safety in people living with muscle-wasting conditions in the UK.

UNITE-DMD is an international collaboration working on gene therapy. Once preclinical studies are complete, it will assess the safety of a gene therapy for Duchenne muscular dystrophy in a phase I/II clinical trial.

To date, no gene therapy trials for muscle-wasting conditions have been undertaken in the UK. The work of UNITE-DMD is therefore fundamental in evaluating the safety of this technique – something that we strongly support.

Although this particular gene therapy will be designed to treat Duchenne muscular dystrophy, its development will refine and improve the technique generally. This will help in the development of gene therapies for other muscle-wasting conditions in the future.

UNITE-DMD is a four-year project taking place in the UK and France. The UK investigators are Professor George Dickson at Royal Holloway, University of London; Professor Francesco Muntoni at the University College London Great Ormond Street Institute of Child Health; and Professor Volker Straub at the John Walton Muscular Dystrophy Research Centre, Newcastle University.

We are delighted to be funding the UK side of the project, in partnership with Action Duchenne. The French Muscular Dystrophy Association (AFM-Telethon) is funding the French arm of the project.

Our work has consistently been supported by funding from MDUK, providing the means to develop these new therapies. This new investment will help to move them into the clinic for the benefit of patients and their families.”

 

Professor George Dickson, who has studied gene therapy for over 20 years and is one of the leaders of the UNITE-DMD project

It’s very important that the charity invests in gene therapy, as this is where the new generation of treatments is going to come from. It might be a game changer for those with our condition. We need to get in on the ground floor of research into gene therapy so we’re not left behind by it.”

 

Baroness Celia Thomas, Muscular Dystrophy UK Trustee, who has limb girdle muscular dystrophy


Gene therapy is intended to be a single treatment for genetic conditions, such as muscular dystrophies. These conditions are caused by a mutation in a gene. The mutated gene does not produce the protein needed to keep the muscle healthy. Gene therapy introduces a new, healthy copy of this gene into the body. This then restores production of the protein that was missing.


Duchenne muscular dystrophy is caused by a faulty dystrophin gene. The UNITE-DMD project will deliver a smaller but functional copy of the dystrophin gene (known as micro-dystrophin) into people with Duchenne muscular dystrophy. This will increase the production of the micro-dystrophin protein in their muscles and could be a potential treatment for the condition.

A significant benefit of this approach is that it is not mutation-specific.


UNITE-DMD will deliver the micro-dystrophin gene by packaging it into viruses called adeno-associated viruses (AAVs) and injecting these into the bloodstream. Once inside the body, the viruses carry the gene into the muscle cells.

By supporting world-leading research, Muscular Dystrophy UK is transforming treatments for all people living with a muscle-wasting condition. Because everyone has the right to live life to the fullest.

 

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