Please use this identifier to cite or link to this item: http://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
Source: 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)

Show full item record
Files in This Item:
File Description SizeFormatAccess SettingsVersion 
Microsoft Word - Cover Page.pdf6.76 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Acknowledgement.pdf12.41 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Summary-final.pdf12.87 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Table of Content.pdf28.71 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - List of Tables.pdf19.04 kBAdobe PDF

OPEN

NoneView/Download
List of Figures.pdf35.39 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - List of Abbreviations.pdf13.34 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Literature Review-final.pdf366.34 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Materials and Methods-final.pdf144.01 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Results-final.pdf514.36 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Discussionconclusion-final.pdf93.84 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - References.pdf45.18 kBAdobe PDF

OPEN

NoneView/Download
Microsoft Word - Appendices.pdf19.6 MBAdobe PDF

OPEN

NoneView/Download

Page view(s)

223
checked on Dec 11, 2017

Download(s)

1,187
checked on Dec 11, 2017

Google ScholarTM

Check


Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.