Work has begun in Professor George Dickson’s lab to refine the gene therapy product; the aim is to ensure a pipeline of improved gene therapy products for use in the future. Preparatory work for the clinical trial is also getting underway with Professors Francesco Muntoni and Volker Straub, starting with the recruitment of the clinical teams that will design and oversee the trial.
Things are also progressing according to plan with respect to production of the vector, which needs to be manufactured in large enough amounts to be used in the clinical trial. This work is being funded and carried out by our French partners AFM and Genethon, but forms an essential part to the UNITE-DMD project.
A summary of the work completed in the first year will be available in November.
Find out more about UNITE-DMD.
Learn more about AAV gene therapy for muscle-wasting conditions
Find out more on MDUK-funded Duchenne research