By making this new grant to Dr D’Alessandro, the Myotubular Trust is now funding three complimentary areas of research and collectively, these grants use three of the available animal models to help in the research and investigation of the disease for better understanding. They address a number of the scientific questions most likely to lead to a therapeutic strategy to treat centronuclear and myotubular myopathy
The three areas of research we are funding now cover:
– Gene replacement therapy studies in the XMTM mouse with Dr Anna Buj Bello (INSERM, France);
– Detailed structural studies of muscle from Labrador dogs and human patients affected by these conditions, to learn more about the physical changes that occur in the muscles with Dr Richard Piercy (Royal Veterinary College, London);
– And this year’s grant to Dr D’Alessandro to understand more precisely the molecular pathway(s) in which 3 of the genes associated with MTM/CNM function.