UNITE-DMD researchers demonstrate efficacy of Duchenne gene therapy

Published Date
Laura Jacobs

Researchers from France and the UK have demonstrated the efficacy of micro-dystrophin gene therapy in a dog model of Duchenne muscular dystrophy. This research is essential for advancing to clinical trials in humans with the condition.

The study was carried out by a collaboration of researchers from Genethon, the AFM-Telethon laboratory, INSERM (UMR 1089, Nantes) and Royal Holloway University of London. Many of these researchers are involved in the UNITE-DMD project that we are co-funding with AFM and Action Duchenne.

This particular gene therapy used an adeno-associated virus (AAV) to deliver a smaller but functional version of the dystrophin gene (called micro-dystrophin). It is not possible to deliver the full-length dystrophin gene as it is too large to fit into an AAV.

The researchers injected the micro-dystrophin gene therapy into the bloodstream of eight dogs naturally affected by Duchenne muscular dystrophy. The dogs were monitored for up to two years after the injection.

The gene therapy stabilised, or at least slowed down, the progression of the condition in most of the dogs. It significantly increased the amount of dystrophin protein in the dogs’ muscles, which led to an improvement in muscle function and gait (manner of walking).

Various tests showed that the micro-dystrophin gene therapy was safe and did not cause serious immune reactions.

Professor George Dickson, one of the leaders of the study and the UNITE-DMD project, said:

This is tremendously exciting progress towards a gene therapy for DMD. The studies in GRMD dogs have been spectacular and exceeded our expectations. My team has worked for many years to optimise a gene therapy medicine for DMD, and now the quite outstanding work of colleagues in France, in Genethon, in Nantes and in Paris has taken us close to clinical trials in DMD patients. I pay thanks also to the amazing and steadfast support of this research by AFM-Telethon and Muscular Dystrophy UK which has been essential to this achievement.

The study was published in the scientific journal, Nature Communications.

Further information

Read the press release from Royal Holloway University

Find out about the UNITE-DMD gene therapy project

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