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Iranian Journal of Basic Medical Sciences، جلد ۲۴، شماره ۸، صفحات ۱۱۵۳-۱۱۵۸
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عنوان انگلیسی Dystrophin gene editing by CRISPR/Cas9 system in human skeletal muscle cell line (HSkMC)
چکیده انگلیسی مقاله Objective(s): Duchene muscular dystrophy (DMD) is a progressive neuromuscular disease caused by mutations in the DMD gene, resulting in the absence of dystrophin expression leading to membrane fragility and myofibril necrosis in the muscle cells. Because of progressive weakness in the skeletal and cardiac muscles, premature death is inevitable. There is no curative treatment available for DMD. In recent years, advances in genetic engineering tools have made it possible to manipulate gene sequences and accurately modify disease-causing mutations. CRISPR/Cas9 technology is a promising tool for gene editing because of its ability to induce double-strand breaks in the DNA. Materials and Methods: In this study for the exon-skipping approach, we designed a new pair of guide RNAs (gRNA) to induce large deletion of exons 48 to 53 in the DMD gene in the human skeletal muscle cell line (HSkMC), in order to correct the frame of the gene.Results: Data showed successful editing of DMD gene by deletion of exons 48 to 53 and correction of the reading frame in edited cells. Despite a large deletion in the edited DMD gene, the data of real-time PCR, immune florescent staining demonstrated successful expression of truncated dystrophin in edited cells.Conclusion: This study demonstrated that the removal of exons 48-53 by the CRISPR / Cas9 system did not alter the expression of the DMD gene due to the preservation of the reading frame of the gene.
کلیدواژه‌های انگلیسی مقاله CRISPR/Cas9, DMD, Dystrophin, Gene editing, HSkMC
نویسندگان مقاله | Mahintaj Dara
Department of Molecular Medicine, School of Advanced Medical Science and Technology, Shiraz University of Medical Science, Shiraz, Iran


| vahid razban
Department of Molecular Medicine, School of Advanced Medical Science and Technology, Shiraz University of Medical Science, Shiraz, Iran


| Mohsen Mazloomrezaei
Student Research Committee, Shiraz University of Medical Science, Shiraz, Iran


| Maryam Ranjbar
Department of Medical Genetics, School of Medicine, Shiraz University of Medical Sciences, Shiraz, Iran


| Marjan Nourigorji
Department of Medical Genetics, School of Medicine, Shiraz University of Medical Sciences, Shiraz, Iran


| Mehdi Dianatpour
Department of Medical Genetics, School of Medicine, Shiraz University of Medical Sciences, Shiraz, Iran|Stem Cell Technology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran
نشانی اینترنتی https://ijbms.mums.ac.ir/article_18498.html
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زبان مقاله منتشر شده en
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نوع مقاله منتشر شده Short Communication
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