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 15 of 15 for “"ZFNs"”.
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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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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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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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In Vivo Genome Editing: Proof of Concept in Neonatal and Adult Mouse Liver
… most genetic diseases. Zinc finger nucleases (ZFNs) are engineered proteins capable of site-directed DNA cleavage, known to increase the efficiency of gene targeting. Our lab has previously demonstrated in vivo gene targeting of the liver using AAV to deliver ZFNs and therapeutic donor in …
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Improving Viral Vectors for Gene Targeting in Gene Therapy
… been achieved using two zinc-finger nucleases (ZFNs) in any AAV serotype. This is significant because the recognition site for I-Sce is not found in the human genome, while ZFNs are designed to specifically bind in or around a gene of interest. Based on the results from the AAV survey and the …
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Genome-Wide Localization And Novel Deposition Pathways Of Histone Variant H3.3 In Embryonic Stem And Neuronal Precursor Cells
… variants, I use designed zinc finger nucleases (ZFNs) to rapidly knock epitope tags into a single allele of the endogenous histone H3.3B gene in mouse ES cells. Genome-wide analysis reveals that H3.3 is enriched in specific patterns at active and repressed genes with high CpG content promoters, …
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Sequence-Specific Gene Correction of Cystic Fibrosis Airway Basal Cells
… sequence-specific zinc finger nucleases (ZFNs) and single-stranded oligo DNA (ssODN) carrying the correcting sequence via electroporation facilitated the correction. The gene-corrected cells upon <em>in vitro </em>differentiation using air-liquid interface showed presence of …
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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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Correction of Cystic Fibrosis-Specific Induced Pluripotent Stem Cells
… cell type. Then, using zinc finger nucleases (ZFNs) to induce a double strand break at the CF loci, we facilitated homologous directed repair of the break by providing a donor molecule that would, upon recombination, correct the disease-conferring mutation. In doing so, we observed an …
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Androgen Receptor and Prostate Cancer Cell Heterogeneity
… LNCaP clones with zinc finger nucleases (ZFNs) and isogenic AR knockout (AR-KO) LNCaP clones with CRISPR-cas9 technology. With the novel genetically matched clones, we demonstrate that the AR<sup>+</sup> and AR-KO clonal LNCaP cells manifest contrasting tumor-regenerating capacities and …
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Modeling 3D Retinogenesis in Mouse Embryonic Stem Cells Following CRISPR-mediated Crx Knockdown
… editing technologies like Zinc Finger Nucleases (ZFNs) or Transcription Activator-like Effector Nucleases (TALENs).</p> <p>In order to overcome current limitations on disease-specific model systems, we developed a protocol for differentiating mouse embryonic stem cells into 3D retinas <em>in …
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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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Engineering programmable nucleases for in vivo and in vitro applications
… Enzymes such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), clustered regularly interspaced short palindromic repeats (CRISPR) associated nucleases, and Argonaute nucleases (Ago) have revolutionized genome engineering by allowing precise and …
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Development and applications of programmable DNA-guided Argonaute-based artificial restriction enzymes
… artificial restriction enzymes (AREs) such as ZFNs, TALENs, or CRISPR-Cas nucleases were developed. While these AREs have longer recognition sequences compared to type II restriction enzymes, they are not able to produce defined sticky ends on DNA molecules or target all desired DNA sequences …