title: Mammalian animal models for Duchenne muscular dystrophy.
abstract: 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 use of animal models plays undoubtedly an important role. Therefore, a number of different mammalian models for DMD have been described. Much knowledge on the molecular mechanisms underlying the disease has arisen from studies in these animals. However, the use of different models does not 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.
authors.affiliation: Biozentrum, University of Basel, Klingelbergstrasse 70, CH-4056 Basel, Switzerland.
authors.forename: Raffaella
authors.initials: R
authors.lastname: Willmann
authors.forename: Stefanie
authors.initials: S
authors.lastname: Possekel
authors.forename: Judith
authors.initials: J
authors.lastname: Dubach-Powell
authors.forename: Thomas
authors.initials: T
authors.lastname: Meier
authors.forename: Markus A
authors.initials: MA
authors.lastname: Ruegg
mesh_terms: D000818:Animals; D002415:Cats; D004195:Disease Models, Animal; D004285:Dogs; D004353:Drug Evaluation, Preclinical / Q000379:methods / Q000592:standards / Q000639:trends; D055786:Gene Knockout Techniques / Q000379:methods; D006801:Humans; D008322:Mammals / Q000235:genetics / Q000378:metabolism; D051379:Mice; D009124:Muscle Proteins / Q000235:genetics* / Q000378:metabolism*; D018482:Muscle, Skeletal / Q000378:metabolism* / Q000473:pathology / Q000503:physiopathology; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000378:metabolism* / Q000503:physiopathology; D013045:Species Specificity
chemical_list: D009124:Muscle Proteins