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.
Results
Showing 1 to 8 of 8 for “"base-editor"”.
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CRISPR gene editing for Cystic Fibrosis: targeting the G542X mutation with base editor engineered virus-like particles
… care and face a reduced life expectancy. CRISPR-based gene editing offers the potential for a cure, by addressing the underlying cause of the disease. The doctoral work presented in this dissertation explored the avenue of CRISPR therapeutic gene editing for CF, while also testing and …
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Functional assessment of BRAF and CRAF through Base Editor 3 mutagenesis reveals residues and regions critical for ERK1/2 signalling
… drive paradoxical activation of wild-type RAF. Base editor 3 (BE3) is a gene editing tool comprised of a nickase Cas9 fused to a cytidine deaminase and a uracil DNA glycosylase inhibitor. This can be used to generate high-efficiency C to T mutations. This thesis describes the use of a high …
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Base editing of Galactose-1-Phosphate Uridylyl Transferase as a novel gene therapy approach to treat Q188R mutation in a cellular model of Classic Galactosemia
… galactitol. The Q188R mutation is caused by a base change from adenine to guanine in base pair 563. This mutation causes an amino acid change in position 188 from glutamine (Gln) to arginine (Arg) and is responsible for 60-70% of CG cases in homozygous state. Consequently, affected infants …
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GATA1 Occupancy Site Regulates Erythroid Cell Fitness through Pleiotropic Functions
… to phenotype. In this study, we combined base editor-mediated perturbations of regulatory elements, CRISPR-mediated gene disruptions, epigenetic profiling, and chromatin organization data with hybrid deep learning models to quantitatively predict the functional consequences of mutations …
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An expert system for off-line analysis of rotating equipment
… proof for the proposed cause the difficulty. An editor has also been built which will allow an expert to keep the information used by the system current with the state of the art for rotating machinery diagnostics. This thesis documents the development of the PC-based Turbo Prolog expert shell …
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Quantitative modelling of CRISPR-Cas editing outcomes
… of individual genetic outcomes generated by each editor. This variance is reproducible and a function of the sequence being targeted, thus making it amenable to quantitative modelling. My research focuses on building such models in a variety of contexts. In this thesis, I recount the history of …
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CRISPR Cas9 targeted single nucleotide change at the (+15) loci of the alpha-lactalbumin gene
Submission published under a 24 month embargo labeled 'U of I Access', the embargo will last until 2024-08-01
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Understanding information at the biomolecular level using statistics and machine learning
Submission original under an indefinite embargo labeled 'Open Access'. The submission was exported from vireo on 2022-11-11 without embargo terms