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 18 of 18 for “"Zinc-finger nucleases"”.
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Optimizing Zinc Finger Nucleases for Use in Mammalian Cells
… a double strand break 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 …
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The Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus
… work, we describe the engineering of artificial zinc finger transcription 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 …
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THE ROLE OF DMRT GENES DURING NEUROGENESIS IN THE ZEBRAFISH FOREBRAIN
Dmrt genes encode zinc finger transcription factors with important roles in sexual development as well as cell fate decisions in non-gonadal tissues. I aimed to analyse the roles of dmrt3 and dmrt5 during forebrain development in zebrafish. Here I show that dmrt5 is a critical regulator of neuronal …
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Využitie transgénnych rastlín pri štúdiu funkcie rastlinných génov
… particular selected gene using site-specific endonucleases, which include meganucleases, zinc finger nucleases, TALEN (transcription activator-like effector nucleases) and CRISPR (clustered regularly interspaced short paindromic repeats). In contrast to inactivation approaches, there are …
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Sequence-Specific Gene Correction of Cystic Fibrosis Airway Basal Cells
… for CF. Delivery of engineered 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 …
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Correction of Cystic Fibrosis-Specific Induced Pluripotent Stem Cells
… to differentiate into any 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, …
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Androgen Receptor and Prostate Cancer Cell Heterogeneity
… RFP-tagged AR<sup>+</sup> 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 …
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Modeling 3D Retinogenesis in Mouse Embryonic Stem Cells Following CRISPR-mediated Crx Knockdown
… than other genome 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 …
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Cystic fibrosis gene repair: correction of ΔF508 using ZFN and CRISPR/Cas9 guide RNA gene editing tools
… This thesis investigates whether existing zinc finger nucleases designed in this lab and CRISPR/gRNAs designed in this thesis can mediate efficient homology-directed repair (HDR) with appropriate donor repair plasmids to correct CF-causing mutations in a CF cell line. Firstly, the most …
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Applications of mitochondrial gene therapy
… diseases. Engineered mitochondrially targeted zinc finger nucleases (mtZFNs) have been successfully used to selectively degrade mutation bearing mtDNA both *in vivo* and *in vitro*, resulting in a shift in the genetic makeup of affected mitochondria and subsequently to phenotypic rescue. Due to …
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Gene Targeting in a Novel Mouse Model and the Chicken DT40 Cell Line
… These breaks can be created 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 …
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Genome-Wide Localization And Novel Deposition Pathways Of Histone Variant H3.3 In Embryonic Stem And Neuronal Precursor Cells
… Hira. To distinguish H3 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 …
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In Vivo Genome Editing: Proof of Concept in Neonatal and Adult Mouse Liver
… of therapeutic value for 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 …
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The Neuropeptide Regulation of Host-Seeking Behavior in Aedes Aegypti Mosquitoes
… we pioneered targeted mutagenesis with zinc-finger nucleases to create multiple NPYLR1 null-mutant mosquito lines. We predicted that these mutants would no longer show inhibition of host-seeking behavior after a blood meal. While we can say with certainty that NPYLR1 is a receptor for …
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Improving Viral Vectors for Gene Targeting in Gene Therapy
… targeting has never 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 …
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Engineering programmable nucleases for in vivo and in vitro applications
Programmable nucleases are powerful molecular tools that have significantly advanced biotechnology development. Enzymes such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), clustered regularly interspaced short palindromic repeats (CRISPR) associated …
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Mitochondrial genome engineering in the murine germline using designer nuclease technology
… binding domains, consisting of either a dimeric zinc finger protein (ZFP) or a transcription activator-like effector (TALE) domain, is conjugated to a dimeric nuclease domain, producing zinc finger nucleases (ZFN) or TALE-nucleases (TALEN), respectively. By targeting these pairs of engineered …
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Genetic Correction of Duchenne Muscular Dystrophy using Engineered Nucleases
… the native dystrophin gene using engineered nucleases that target one or more exons in a mutational hotspot in exons 45-55 of the dystrophin gene. Importantly, this hotspot mutational region collectively represents approximately 62% of all DMD mutations. In this work, we utilize various …