Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder caused by inability to produce dystrophin protein. In most patients, the expression of dystrophin lost due to disrupting mutations in open reading frame. Despite the efforts in a large number of different therapeutic...
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| Format: | Article |
| Language: | English |
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KeAi Communications Co., Ltd.
2021-03-01
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| Series: | Genes and Diseases |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2352304220300015 |
| _version_ | 1797715184160079872 |
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| author | Hasan Mollanoori Yazdan Rahmati Bita Hassani Meysam Havasi Mehr Shahram Teimourian |
| author_facet | Hasan Mollanoori Yazdan Rahmati Bita Hassani Meysam Havasi Mehr Shahram Teimourian |
| author_sort | Hasan Mollanoori |
| collection | DOAJ |
| description | Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder caused by inability to produce dystrophin protein. In most patients, the expression of dystrophin lost due to disrupting mutations in open reading frame. Despite the efforts in a large number of different therapeutic approaches to date, the treatments available for DMD remain mitigative and supportive to improve the symptoms of the disease, rather than to be curative. The advent of CRISPR/Cas9 technology has revolutionized genome editing scope and considered as pioneer in effective genomic engineering. Deletions or excisions of intragenic DNA by CRISPR as well as a similar strategy with exon skipping at the DNA level induced by antisense oligonucleotides, are new and promising approaches in correcting DMD gene, which restore the expression of a truncated but functional dystrophin protein. Also, CRISPR/Cas9 technology can be used to treat DMD by removing duplicated exons, precise correction of causative mutation by HDR-based pathway and inducing the expression of compensatory proteins such as utrophin. In this study, we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD. |
| first_indexed | 2024-03-12T08:03:09Z |
| format | Article |
| id | doaj.art-9bf6e940b10a4f5aaec4904f8baddeb6 |
| institution | Directory Open Access Journal |
| issn | 2352-3042 |
| language | English |
| last_indexed | 2024-03-12T08:03:09Z |
| publishDate | 2021-03-01 |
| publisher | KeAi Communications Co., Ltd. |
| record_format | Article |
| series | Genes and Diseases |
| spelling | doaj.art-9bf6e940b10a4f5aaec4904f8baddeb62023-09-02T19:44:43ZengKeAi Communications Co., Ltd.Genes and Diseases2352-30422021-03-0182146156Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophyHasan Mollanoori0Yazdan Rahmati1Bita Hassani2Meysam Havasi Mehr3Shahram Teimourian4Department of Medical Genetics, Iran University of Medical Sciences (IUMS), Tehran, 1449614535, IranDepartment of Medical Genetics, Iran University of Medical Sciences (IUMS), Tehran, 1449614535, IranDepartment of Medical Genetics, Shahid Beheshti University of Medical Sciences (SBMU), Tehran, 1985717443, IranDepartment of Physiology, Iran University of Medical Sciences (IUMS), Tehran, 1449614535, IranDepartment of Medical Genetics, Iran University of Medical Sciences (IUMS), Tehran, 1449614535, Iran; Corresponding author.Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder caused by inability to produce dystrophin protein. In most patients, the expression of dystrophin lost due to disrupting mutations in open reading frame. Despite the efforts in a large number of different therapeutic approaches to date, the treatments available for DMD remain mitigative and supportive to improve the symptoms of the disease, rather than to be curative. The advent of CRISPR/Cas9 technology has revolutionized genome editing scope and considered as pioneer in effective genomic engineering. Deletions or excisions of intragenic DNA by CRISPR as well as a similar strategy with exon skipping at the DNA level induced by antisense oligonucleotides, are new and promising approaches in correcting DMD gene, which restore the expression of a truncated but functional dystrophin protein. Also, CRISPR/Cas9 technology can be used to treat DMD by removing duplicated exons, precise correction of causative mutation by HDR-based pathway and inducing the expression of compensatory proteins such as utrophin. In this study, we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD.http://www.sciencedirect.com/science/article/pii/S2352304220300015CRISPR/Cas9Duchenne muscular dystrophyGenome editingTherapeutic approachesX-linked recessive |
| spellingShingle | Hasan Mollanoori Yazdan Rahmati Bita Hassani Meysam Havasi Mehr Shahram Teimourian Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy Genes and Diseases CRISPR/Cas9 Duchenne muscular dystrophy Genome editing Therapeutic approaches X-linked recessive |
| title | Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy |
| title_full | Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy |
| title_fullStr | Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy |
| title_full_unstemmed | Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy |
| title_short | Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy |
| title_sort | promising therapeutic approaches using crispr cas9 genome editing technology in the treatment of duchenne muscular dystrophy |
| topic | CRISPR/Cas9 Duchenne muscular dystrophy Genome editing Therapeutic approaches X-linked recessive |
| url | http://www.sciencedirect.com/science/article/pii/S2352304220300015 |
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