Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an incurable disease and the search for a cure is a challenging journey. However, with recent encouraging progress, we are seeing a light at the end of a long tunnel. This review focuses on several main strategies in gene therapy, including truncated dystrophin g...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2014
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| Online Access: | http://psasir.upm.edu.my/id/eprint/37897/ http://psasir.upm.edu.my/id/eprint/37897/1/Hunting%20for%20a%20cure%20the%20therapeutic%20potential%20of%20gene%20therapy%20in%20Duchenne%20muscular%20dystrophy.pdf |
| _version_ | 1848848730278592512 |
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| author | Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Hoo, Fan Kee Basri, Hamidon |
| author_facet | Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Hoo, Fan Kee Basri, Hamidon |
| author_sort | Hashim, Hasnur Zaman |
| building | UPM Institutional Repository |
| collection | Online Access |
| description | Duchenne muscular dystrophy (DMD) is an incurable disease and the search for a cure is a challenging journey. However, with recent encouraging progress, we are seeing a light at the end of a long tunnel. This review focuses on several main strategies in gene therapy, including truncated dystrophin gene transfer via viral vectors, antisense mediated exon skipping to restore the reading frame, and read-through of translation stop codons. An exon skipping agent, eteplirsen, and a termination codon read drug, ataluren, are currently the most promising therapies. With better understanding of the molecular mechanism, gene therapy has improved with regard to the key areas of gene stability, safety, and route of delivery. Consequently, it has emerged as an exciting and hopeful means for novel treatment of this devastating disease. |
| first_indexed | 2025-11-15T09:39:08Z |
| format | Article |
| id | upm-37897 |
| institution | Universiti Putra Malaysia |
| institution_category | Local University |
| language | English |
| last_indexed | 2025-11-15T09:39:08Z |
| publishDate | 2014 |
| publisher | Elsevier |
| recordtype | eprints |
| repository_type | Digital Repository |
| spelling | upm-378972015-12-21T06:25:18Z http://psasir.upm.edu.my/id/eprint/37897/ Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Hoo, Fan Kee Basri, Hamidon Duchenne muscular dystrophy (DMD) is an incurable disease and the search for a cure is a challenging journey. However, with recent encouraging progress, we are seeing a light at the end of a long tunnel. This review focuses on several main strategies in gene therapy, including truncated dystrophin gene transfer via viral vectors, antisense mediated exon skipping to restore the reading frame, and read-through of translation stop codons. An exon skipping agent, eteplirsen, and a termination codon read drug, ataluren, are currently the most promising therapies. With better understanding of the molecular mechanism, gene therapy has improved with regard to the key areas of gene stability, safety, and route of delivery. Consequently, it has emerged as an exciting and hopeful means for novel treatment of this devastating disease. Elsevier 2014-03 Article PeerReviewed application/pdf en http://psasir.upm.edu.my/id/eprint/37897/1/Hunting%20for%20a%20cure%20the%20therapeutic%20potential%20of%20gene%20therapy%20in%20Duchenne%20muscular%20dystrophy.pdf Hashim, Hasnur Zaman and Che Abdullah, Shahrin Tarmizi and Wan Sulaiman, Wan Aliaa and Hoo, Fan Kee and Basri, Hamidon (2014) Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy. Tzu Chi Medical Journal, 26 (1). pp. 5-9. ISSN 1016-3190 http://www.sciencedirect.com/science/article/pii/S101631901400007X 10.1016/j.tcmj.2014.02.002 |
| spellingShingle | Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Hoo, Fan Kee Basri, Hamidon Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy |
| title | Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy |
| title_full | Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy |
| title_fullStr | Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy |
| title_full_unstemmed | Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy |
| title_short | Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy |
| title_sort | hunting for a cure: the therapeutic potential of gene therapy in duchenne muscular dystrophy |
| url | http://psasir.upm.edu.my/id/eprint/37897/ http://psasir.upm.edu.my/id/eprint/37897/ http://psasir.upm.edu.my/id/eprint/37897/ http://psasir.upm.edu.my/id/eprint/37897/1/Hunting%20for%20a%20cure%20the%20therapeutic%20potential%20of%20gene%20therapy%20in%20Duchenne%20muscular%20dystrophy.pdf |