The Neuroscience Journal of Shefaye Khatam
مجله علوم اعصاب شفای خاتم
Shefaye Khatam
Medical Sciences
http://shefayekhatam.ir
1
admin
2322-1887
2345-4814
10.61186/shefa
fa
jalali
1397
1
1
gregorian
2018
4
1
6
2
online
1
fulltext
en
P164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy
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. 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