News & events News Action Duchenne Funding Gene Therapy to Advance Treatments for Duchenne We are thrilled to announce our funding of “UNITE-DMD”, the first gene therapy trials for Duchenne muscular dystrophy here in the UK. From the very early days of Action Duchenne, we have supported research into gene therapy as a potential way of treating Duchenne muscular dystrophy. This area has seen significant progress in recent years, and now is the time to test its safety in people living with Duchenne, here in the UK. As soon as preclinical studies have been completed, this international collaboration will assess the safety of gene therapy in Duchenne, in a phase I/II clinical trial that will take place in both the UK and France. As these will be the first gene therapy trials for Duchenne in the UK, UNITE-DMD will be vital to test the safety of this technology. UNITE-DMD is a four year project taking place in the UK and France. The UK investigators on the project 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; 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 principal funder Muscular Dystrophy UK. Together we are investing over £1.6m into UNITE-DMD. The French Muscular Dystrophy Association (AFM-Téléthon) is funding the French arm of the project. Edit Professor George Dickson has been studying gene therapy for over 20 years and is one of the key leaders of the UNITE-DMD project: “Our work has been supported by funding from Action Duchenne to develop advanced medicines for DMD gene therapy. This new investment will help move them into the clinic for the benefit of patients and their families”. Action Duchenne CEO, Diana Ribeiro says: “Since Action Duchenne was founded in 2001, we and other experts in the field, felt that gene therapy was a state-of-the-art advance for Duchenne translational research. We provided strategic direction and financial support for this potential therapy. Most importantly to those living with Duchenne and their families, this technology is non-mutation specific and could treat a wider range of the community.” Edit What is gene therapy? Gene therapy is intended to be a single treatment for genetic conditions, such as Duchenne muscular dystrophy. 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. How does this work for Duchenne muscular dystrophy? 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. How does this gene get into the body? 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. Image by Lucy Reading-Ikkanda for The Scientist (“Targeting DNA,” June 2012) Action Duchenne has always supported the best research into Duchenne from around the world, to improve lives while searching for a cure, for people living with Duchenne. By supporting this innovative translational research we aim to expand the number of people with Duchenne who could potentially benefit from the advances in Duchenne research. We will be sharing more information on the trial in the coming months so please keep an eye on our website, weekly e-shot and social media. As ever the best place in the UK to hear updates on research, and gain access to experts in Duchenne care, is at the annual Action Duchenne International Conference. The 2017 event is 10-12 November at the Hilton Birmingham Metropole so please put the date in your diary. We would like to take this opportunity to thank all of our amazing supporters for their hard work, tenacity and generosity. Without you, funding ground-breaking projects like UNITE-DMD, would not be possible. We look forward to sharing more information about the project with you soon.