Background Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due to mutations in the dystrophin gene. Animal models that accurately reflect pathological conditions and disease characteristics are key factors in the discovery and development of new anti-DMD drugs. Aim Here, we evaluated motor behavior, pathological and biochemical characters of a new DMD mouse model built up by the Nanjing Biomedical Research Institute of Nanjing University (NBRI). Methods The pole test and open-field test were used to assess the movement disorders in DMD mouse model. The gastrocnemius (GAS), biceps, triceps, soleus, and tibialis anterior muscles of mice were subjected to weight analysis to evaluate the skeletal muscle pseudohypertrophy. Meanwhile, immunofluorescence and Western blotting were used to detect the expression of dystrophin in the GAS. Serum levels of creatine kinase (CK) and lactate dehydrogenase (LDH) that accurately reflect muscle damage were detected. Masson staining was used to evaluate the fibrosis of GAS and diaphragm (DIA). Results The novel DMD mouse showed significant behavioral disorders and exhibited high serum levels of CK and LDH. Western blotting and immunofluorescence staining showed decreased significantly with dystrophin level in the GAS. Besides, the mdx mouse of DMD developed fibrosis in both GAS and DIA. Conclusion Taken together, our results indicated that the behavioral, biochemical and pathological characterization of the mdx mouse model is similar to human DMD. This mdx mouse model may provide insights into the pathophysiology of DMD and the effects of anti-DMD drugs.

Core tip: Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due to mutations in the dystrophin gene. Animal models that accurately reflect pathological conditions and disease characteristics are key factors in the discovery and development of new anti-DMD drugs. Here, we described a novel DMD mouse model built up by the Nanjing Biomedical Research Institute of Nanjing University (NBRI). We found that the DMD mouse showed significant behavioral disorders and exhibited increased serum creatine kinase (CK) and lactate dehydrogenase. Western blotting and Immunofluorescence staining also showed significantly decreased expression level of dystrophin in the gastrocnemius (GAS) muscle. Besides, the mdx mouse of DMD developed fibrosis in both GAS and diaphragm (DIA). Taken together, our results indicated that the behavioral, biochemical and pathological characterization of the mdx mouse model is consistent with human DMD. This genetic mouse model may provide insights into the pathophysiology of DMD and the effects of anti-DMD drugs.