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Bioinformatics design of cis-regulatory elements controlling human gene expression Farkas, Rachelle
Abstract
Gene therapy has the potential to not only treat, but cure individuals suffering from inherited diseases. Advances in understanding the human genome and the discovery of causal genes underlying diseases has heightened the need to solve the gene therapy challenge. Viral vectors are often used as a delivery tool for therapeutics, but their safety and efficacy are still being studied. To contribute to this goal, we have created 49 small viral promoters by bioinformatically annotating cis-regulatory regions from which a subset are concatenated with the goal of driving cell-specific expression of a reporter gene. We have tested a subset of these in mice in vivo. Regulatory region analysis can take a trained designer multiple weeks. To resolve this issue, we have created a semi-automated approach to regulatory region identification, named OnTarget. The OnTarget database accumulates thousands of cell and tissue-specific experiments in order to identify regions informative of regulatory properties. OnTarget is able to identify regulatory regions consistent with those identified by designers. In this capacity, we expect OnTarget to lead to better and faster identification of cis-regulatory regions for the design of promoters targeting specific sets of cells.
Item Metadata
Title |
Bioinformatics design of cis-regulatory elements controlling human gene expression
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Creator | |
Publisher |
University of British Columbia
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Date Issued |
2017
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Description |
Gene therapy has the potential to not only treat, but cure individuals suffering from inherited diseases. Advances in understanding the human genome and the discovery of causal genes underlying diseases has heightened the need to solve the gene therapy challenge. Viral vectors are often used as a delivery tool for therapeutics, but their safety and efficacy are still being studied. To contribute to this goal, we have created 49 small viral promoters by bioinformatically annotating cis-regulatory regions from which a subset are concatenated with the goal of driving
cell-specific expression of a reporter gene. We have tested a subset of these in mice in vivo. Regulatory region analysis can take a trained designer multiple weeks. To resolve this issue, we have created a semi-automated approach to regulatory region identification, named OnTarget. The OnTarget database accumulates thousands of cell and tissue-specific experiments in order to identify regions informative of regulatory properties. OnTarget is able to identify regulatory regions consistent with those identified by designers. In this capacity, we expect OnTarget to lead to better
and faster identification of cis-regulatory regions for the design of promoters targeting specific sets of cells.
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Genre | |
Type | |
Language |
eng
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Date Available |
2018-10-31
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Provider |
Vancouver : University of British Columbia Library
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Rights |
Attribution-NonCommercial-NoDerivatives 4.0 International
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DOI |
10.14288/1.0357223
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URI | |
Degree | |
Program | |
Affiliation | |
Degree Grantor |
University of British Columbia
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Graduation Date |
2017-11
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Campus | |
Scholarly Level |
Graduate
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Rights URI | |
Aggregated Source Repository |
DSpace
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Rights
Attribution-NonCommercial-NoDerivatives 4.0 International