Genome engineering using DNA-binding proteins: zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs)

dc.contributor.advisor Drena Dobbs
dc.contributor.advisor Edward Yu Reyon, Deepak
dc.contributor.department Genetics, Development and Cell Biology 2018-08-11T19:17:47.000 2020-06-30T02:58:52Z 2020-06-30T02:58:52Z Sat Jan 01 00:00:00 UTC 2011 2001-01-01 2011-01-01
dc.description.abstract <p>Over the past two decades, research groups in both academia and private industry have developed key technologies, including viral delivery vectors and engineered transposon-based or zinc finger protein-based nucleases, towards achieving the long-sought goal of therapeutic genome editing in humans. To date, Zinc Finger Nucleases (ZFNs) have been the most promising reagents for potential therapeutic applications in humans, but the recently characterized Transcription Activator Like Effector (TALE) proteins may soon change this status quo. Although it remains to be seen whether nucleases based on these proteins (TALENs) will be as broadly applicable and effective as ZFNs, based on initial reports, TALENs look very promising. Currently, the primary advantage of TALENs is that the DNA binding code for TALENs appears to be simple and robust, making their synthesis relatively simple.</p> <p>In this dissertation, I summarize advances made in the field of genome editing over the past decade and compare and contrast the currently available tools, focusing on ZFNs and TALENs. Specifically, I describe our efforts to make ZFN technology more accessible by designing and implementing models to help researchers choose target sites that are most amenable to targeting using ZFNs. Also, to help explore the potential of TALENs as tools for genome editing, I describe the development of a simple protocol to aid in constructing TALENs. As ZFNs become easier to use, and TALENs become more robust, the use of genome editing techniques as therapeutics appears poised to become reality in the near future.</p>
dc.description.comments <p>ISBN: 9781267153203<strong></strong></p>
dc.format.mimetype application/pdf
dc.identifier archive/
dc.identifier.articleid 5752
dc.identifier.contextkey 8098942
dc.identifier.s3bucket isulib-bepress-aws-west
dc.identifier.submissionpath etd/14745
dc.language.iso en
dc.source.bitstream archive/|||Fri Jan 14 20:25:50 UTC 2022
dc.subject.disciplines Bioinformatics
dc.subject.disciplines Computational Biology
dc.subject.disciplines Genomics
dc.subject.keywords Bioinformatics and Computational Biology
dc.subject.keywords Biological Sciences
dc.subject.keywords Zinc finger nucleases
dc.subject.keywords Transcription activator
dc.subject.keywords Nucleases
dc.subject.keywords DNA-binding proteins
dc.title Genome engineering using DNA-binding proteins: zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs)
dc.type article
dc.type.genre dissertation
dspace.entity.type Publication
relation.isOrgUnitOfPublication 9e603b30-6443-4b8e-aff5-57de4a7e4cb2 Bioinformatics and Computational Biology dissertation Doctor of Philosophy
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