Many aspects of gene therapy in duchenne muscular dystrophy

IntroductionDuchenne muscular dystrophy (DMD), is the most common sever life threatening form of muscular dystrophy especially in childhood. DMD is an X-linked, inherited muscular disease that is caused by recessive and monogenic mutation in the dystrophin gene. The symptoms are presented by progressive muscle degeneration, weakness, leading to premature death in adulthood. About 1 in 5000 newborn boys, are suffering from DMD.ObjectivesThis study aimed to investigate the Duchenne muscular dystrophy gene therapy, evaluating DMD, other types of MDs, suitable vectors and results of animal tests.MethodsWe conducted a literature review about DMD gene therapy from 2008-2018 time interval, using concise keywords, scientific search engines and databases such as Google Scholar, Scopus, Academic Search, PubMed, Springer, SID, Iran doc. Initially, 74 articles were founded and then, totally 33 article were selected for this study.ResultsStudies on Adeno-associated virus (AAV) as the common vector used for this therapy and researches showed that synthetic AAV minidystrophin gene is effective for animal models such as mdx mouse. In most of researches, intramuscular injection of minidystrophin transgene caused restoring dystrophin and dystrophin-associated complex, ameliorated dystrophic pathology and led cell to normal morphology and histology features. A newer article also shows injecting dual AAV vectors into canine models of DMD, diminishes muscle degeneration and fibrosis and also improves myofiber size distribution.ConclusionSince most of researches show the benefits of gene therapy for treatment of DMD, and whole body systemic gene therapy is likely the most effective way to lessen greatly the disease burden of DMD; the researches on animal models should be continued till finding the most suitable treatment in human

کلیدواژه ها:

Gene therapy ، Duchenne muscular dystrophy