en P164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy تحقیقات پایه در علوم اعصاب Basic research in Neuroscience مروری Review --- Open Access, CC-BY-NC <div style="text-align: center;"><span style="background:#FBFAF9;"><span style="color:black;"><span style="font-family:tahoma,sans-serif;"><span style="font-size:9.0pt;">لطفاً به چکیده انگلیسی مراجعه شود</span></span></span></span><span dir="LTR" style="background:#FBFAF9;"><span style="color:black;"><span style="font-family:tahoma,sans-serif;"><span style="font-size:9.0pt;">.</span></span></span></span></div> <div style="text-align: justify;">Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate candidate for this strategy to restore dystrophin production in most affected tissues. This review has focused on elucidating the role of Adeno-associated viral vectors in duchenne muscle dystrophy. Some strategies in gene therapy of BMD exon skipping, protein upregulation, stem cell transplants and mutation suppression in order to restore dystrophin production. Serious adverse events have been limited them. &nbsp;One of the novel and functional strategy to replace dystrophin is using shuttle vectors derived from adeno-associated virus (AAV). This method has been tested in numerous human clinical trials without life threatening adverse effects. Major limitations of AAV vectors include limited cloning capacity and activation of immune response. Therefore, using miniaturized dystrophin and effective methods in order to attenuate immune system can promote this strategy.</div> Duchenne, Muscular Dystrophy, Adeno-Associated Viral (AAV) Vectors, Gene Therapy 195 195 http://shefayekhatam.ir/browse.php?a_code=A-10-24-1226&slc_lang=en&sid=1 Zahra Behrooznia Zahra Behrooznia z.behrooznia@gmail.com 100319475328460016244 100319475328460016244 Yes Student Research Committee, Faculty of Medicine, Islamic Azad University, Mashhad, Iran Student Research Committee, Faculty of Medicine, Islamic Azad University, Mashhad, Iran Shadi Sarafan Shadi Sarafan 100319475328460016245 100319475328460016245 No Student Research Committee, Faculty of Medicine, Islamic Azad University, Mashhad, Iran Student Research Committee, Faculty of Medicine, Islamic Azad University, Mashhad, Iran Mohsen Mahdinejad Kashani Mohsen Mahdinejad Kashani 100319475328460016246 100319475328460016246 No Neurology Department, Faculty of Medicine, Mashhad Branch, Islamic Azad University, Mashhad, Iran Neurology Department, Faculty of Medicine, Mashhad Branch, Islamic Azad University, Mashhad, Iran