Hope for myopathy sufferers

The facts & the project

Dystrophin is a key protein that plays a role in helping muscles resist stress but its exact function, so essential to the survival of the muscle, is still relatively unknown even on the molecular scale, chiefly because of its size. Patients with Duchene and Becker’s muscular dystrophy suffer early deaths as a result of the absence or malfunctioning of dystrophin. Thus, describing and understanding the structure, dynamic and network of interactions of this protein at a molecular level represent a vital stage in optimising genetic treatments that are currently under development, or for in refining new therapeutic approaches.

The results

With the support of AFM-Téléthon, combining experimental approaches and molecular simulations using GENCI’s Ada computer, this work carried out at the University of Rennes 1, made it possible for the first time to apply a dynamic and complete structural model of the central part of the protein (previously only 4% of its total size had been defined) and then associate it with a number of its biomolecular partners.

Details

500 000 core hours on Ada (CNRS/Idris)

Principal Investigator: Olivier Delalande - Université Rennes 1