Please use this identifier to cite or link to this item: 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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