Development of therapy for Duchenne muscular dystrophy.

Abstract

OBJECTIVE: To review and summarize the latest development of the therapy for the Duchenne muscular dystrophy (DMD). METHODS: The recently-published articles related to the therapies for DMD were extensively reviewed and briefly summarized. RESULTS: The therapeutic approaches for DMD included the gene therapy, the cell therapy, and the pharmacological therapy. The gene therapy and the cell therapy were focused on the treatment for the cause of DMD by the delivery of the missing gene, the modification of the mutated gene, and the transfer of the normal cells including the stem cells, while the pharmacological therapy dealt with the downstream events caused by the dystrophin gene defect, slowed down the pathologic progress of DMD, and improved the DMD patient's life quality and life span, by medication and other factor treatments. CONCLUSION: There is still no cure for DMD because of various difficulties in replacing or repairing the defected gene and of the multifaceted nature of the severe symptoms. Therefore, it is imperative for us to find out a more effective treatment that can solve these problems.