Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 8 of 8 for “"ZFN"”.
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Cystic fibrosis gene repair: correction of ΔF508 using ZFN and CRISPR/Cas9 guide RNA gene editing tools
… ΔF508, was corrected using a pair of existing ZFNs, which cleave in intron 9, and the donor repair plasmid pITR-donor-XC, which contains the correct CTT sequence and two unique restriction sites. HDR was initially determined to be <1% but further analysis by next generation sequencing (NGS) …
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The Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus
… factors (ZFTFs) and zinc finger nucleases (ZFNs) to target sequences at the hTERT promoter. We first explored expansions to the repertoire of sites that can be targeted by ZFNs and modifications of ZFN architecture to accommodate such sites. A ZFN is made of a zinc-finger DNA binding domain …
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Optimizing Zinc Finger Nucleases for Use in Mammalian Cells
… within the target locus. Zinc Finger Nucleases (ZFNs) are designed proteins that fuse a zinc finger DNA binding domain to the nuclease domain from the FokI restriction endonuclease and have been used to induce double strand breaks at precise sequences. Although ZFNs have been successfully used to …
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Gene Targeting in a Novel Mouse Model and the Chicken DT40 Cell Line
… by proteins called zinc finger nucleases (ZFNs). ZFN mediated gene targeting is a powerful technology, but has not yet been fully characterized in primary cells. Furthermore, before clinical use in the treatment of monogenic diseases, it is necessary to first test this technology in animal …
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Natural and Exogenous Genome Editing In Wiskott-Aldrich Syndrome Patient Cells
… vitro</em> virus-free zinc-finger nuclease (ZFN)-mediated genome editing strategy for correction of <em>WAS</em> muations. We generated induced pluripotent stem cells (iPSC) from skin fibroblasts of a WAS patient carrying an insertional frame-shift mutation. Subsequently, a WAS-2A-eGFP …
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Genetic Correction of Duchenne Muscular Dystrophy using Engineered Nucleases
… of exons by using zinc-finger nucleases (ZFNs) to selectively remove sequences important in specific exon recognition. This strategy has the advantage of creating predictable frame restoration and protein expression, although it relies on simultaneous nuclease activity to generate genomic …
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Homologous Recombination Outcomes of Double-Strand Break- initiated Events in Saccharomyces cerevisiae
… made by I-SceI versus a Zinc Finger Nuclease (ZFN)-generated break with a 5’ overhang. Results of this study indicate the method of DSB induction does not drastically affect HR outcomes. Although these different initiating-breaks produced a slight difference in the distributions of HR products, …
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Mitochondrial genome engineering in the murine germline using designer nuclease technology
… domain, producing zinc finger nucleases (ZFN) or TALE-nucleases (TALEN), respectively. By targeting these pairs of engineered nucleases adjacent to a site of interest, a DNA double-strand break (DSB) can be produced, leading to degradation of the mtDNA molecule. Using novel architecture to …