Advance and cogitation of gene therapy for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is the common hereditary muscular disease caused by the deficiency of cytoskeletal protein dystrophin on the sarcolemma. It is characterized by progessive muscle weakness and atrophy and dying of heart or respiratory failure. Currently the gene therapy strategies of...
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| Format: | Article |
| Language: | English |
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Tianjin Huanhu Hospital
2019-05-01
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| Series: | Chinese Journal of Contemporary Neurology and Neurosurgery |
| Subjects: | |
| Online Access: | http://cjcnn.org/index.php/cjcnn/article/view/1951 |
| _version_ | 1818025563902705664 |
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| author | Cheng ZHANG Jin-fu LIN Zi-yu LIAO |
| author_facet | Cheng ZHANG Jin-fu LIN Zi-yu LIAO |
| author_sort | Cheng ZHANG |
| collection | DOAJ |
| description | Duchenne muscular dystrophy (DMD) is the common hereditary muscular disease caused by the deficiency of cytoskeletal protein dystrophin on the sarcolemma. It is characterized by progessive muscle weakness and atrophy and dying of heart or respiratory failure. Currently the gene therapy strategies of DMD can be catagorized into two groups: restoring dystrophin expression and compensating for the lack of dystrophin. Therapies restoring dystrophin include nonsense mutation readthrough, exon skipping, adeno-associated virus (AAV) mediated micro-dystrophin therapy and gene editing. Here we summarize the latest advance of gene therapy for DMD, focusing on strategies that restore dystrophin, hoping to benefit the choosing of optimal gene therapy.
DOI: 10.3969/j.issn.1672-6731.2019.05.004 |
| first_indexed | 2024-12-10T04:18:06Z |
| format | Article |
| id | doaj.art-56e37e6c19d54108817654861c4f3c9d |
| institution | Directory Open Access Journal |
| issn | 1672-6731 |
| language | English |
| last_indexed | 2024-12-10T04:18:06Z |
| publishDate | 2019-05-01 |
| publisher | Tianjin Huanhu Hospital |
| record_format | Article |
| series | Chinese Journal of Contemporary Neurology and Neurosurgery |
| spelling | doaj.art-56e37e6c19d54108817654861c4f3c9d2022-12-22T02:02:30ZengTianjin Huanhu HospitalChinese Journal of Contemporary Neurology and Neurosurgery1672-67312019-05-011953123191906Advance and cogitation of gene therapy for Duchenne muscular dystrophyCheng ZHANG0Jin-fu LIN1Zi-yu LIAO2Department of Neurology, the First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, Guangdong, ChinaDepartment of Neurology, the First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, Guangdong, ChinaDepartment of Neurology, the First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, Guangdong, ChinaDuchenne muscular dystrophy (DMD) is the common hereditary muscular disease caused by the deficiency of cytoskeletal protein dystrophin on the sarcolemma. It is characterized by progessive muscle weakness and atrophy and dying of heart or respiratory failure. Currently the gene therapy strategies of DMD can be catagorized into two groups: restoring dystrophin expression and compensating for the lack of dystrophin. Therapies restoring dystrophin include nonsense mutation readthrough, exon skipping, adeno-associated virus (AAV) mediated micro-dystrophin therapy and gene editing. Here we summarize the latest advance of gene therapy for DMD, focusing on strategies that restore dystrophin, hoping to benefit the choosing of optimal gene therapy. DOI: 10.3969/j.issn.1672-6731.2019.05.004http://cjcnn.org/index.php/cjcnn/article/view/1951Muscular dystrophy, DuchenneGenetic therapyReview |
| spellingShingle | Cheng ZHANG Jin-fu LIN Zi-yu LIAO Advance and cogitation of gene therapy for Duchenne muscular dystrophy Chinese Journal of Contemporary Neurology and Neurosurgery Muscular dystrophy, Duchenne Genetic therapy Review |
| title | Advance and cogitation of gene therapy for Duchenne muscular dystrophy |
| title_full | Advance and cogitation of gene therapy for Duchenne muscular dystrophy |
| title_fullStr | Advance and cogitation of gene therapy for Duchenne muscular dystrophy |
| title_full_unstemmed | Advance and cogitation of gene therapy for Duchenne muscular dystrophy |
| title_short | Advance and cogitation of gene therapy for Duchenne muscular dystrophy |
| title_sort | advance and cogitation of gene therapy for duchenne muscular dystrophy |
| topic | Muscular dystrophy, Duchenne Genetic therapy Review |
| url | http://cjcnn.org/index.php/cjcnn/article/view/1951 |
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