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https://scholarbank.nus.edu.sg/handle/10635/23208
Title: | Gene repair in duchenne muscular dystrophy | Authors: | CHAN WEI LOONG | Keywords: | Gene Repair in Duchenne Muscular Dystrophy | Issue Date: | 4-Jun-2007 | Citation: | CHAN WEI LOONG (2007-06-04). Gene repair in duchenne muscular dystrophy. ScholarBank@NUS Repository. | Abstract: | This study seeks to demonstrate gene repair in DMD gene deletion by exon insertion/replacement mediated by RNA splicing. A mini-gene target simulating a mutation model of exon 60 deletion of dystrophin was constructed. Six pre-therapeutic molecules (PTMs) consisting of a 5a?? and 3a?? binding domain, trans-splicing domain and WT replacement exons of 60 and 61 were artificially constructed.The results showed that WT exon 60 was inserted successfully and the trans-splicing efficiency ranged from 10% to 44% by semi quantitative assay. Furthermore, comparison between the six different designs of PTMs showed that masking of endogenous splicing elements of the target mini-gene transcript was critical. Variations in the length of the binding domains were less effective in inducing trans-splicing as compared to the role of the splicing elements. The findings from this study suggest that this approach of trans-splicing mediated exon insertion/replacement may provide a novel strategy for DMD gene repair. | URI: | http://scholarbank.nus.edu.sg/handle/10635/23208 |
Appears in Collections: | Master's Theses (Open) |
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Microsoft Word - Cover Page.pdf | 6.76 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Acknowledgement.pdf | 12.41 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Summary-final.pdf | 12.87 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Table of Content.pdf | 28.71 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - List of Tables.pdf | 19.04 kB | Adobe PDF | OPEN | None | View/Download | |
List of Figures.pdf | 35.39 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - List of Abbreviations.pdf | 13.34 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Literature Review-final.pdf | 366.34 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Materials and Methods-final.pdf | 144.01 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Results-final.pdf | 514.36 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Discussionconclusion-final.pdf | 93.84 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - References.pdf | 45.18 kB | Adobe PDF | OPEN | None | View/Download | |
Microsoft Word - Appendices.pdf | 19.6 MB | Adobe PDF | OPEN | None | View/Download |
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