New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

Evry (France), 5 April, 2017. A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease.
This work, published today in Molecular Therapy, has been achieved thanks to donations from the French Telethon and the support of the Myotubular Trust.