Mammalian animal models for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disease that affects boys and leads to early death. In the quest for new treatments that improve the quality Of life and in the search for a possible definitive cure, the rise of animal models Plays undoubtedly in important role. Therefore, a number of different mammalian models for DMD have been described. Much knowledge oil the molecular mechanisms Underlying the disease has arisen from studies in these animals, However, the rise of different models does nor often allow a direct comparison of results obtained in preclinical trials and therefore hinders a straightforward translational research. In the frame of TREAT-NMD, a European Network of Excellence addressing the fragmentation in the assessment and treatment of neuromuscular diseases. we compare here the currently used mammalian animal models for DMD with the aim of selecting and recommending the most appropriate ones for preclinical efficacy testing of new therapeutic strategies. (C) 2008 Elsevier B.V. All rights reserved.