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 10 of 10 for “"Base Editors"”.
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Targeting Duchenne muscular dystrophy with CRISPR base editors
Submission published under a 24 month embargo labeled 'Closed Access', the embargo will last until 2025-12-01
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Exon skipping therapy for Duchenne Muscular Dystrophy with CRISPR base editors
Submission published under a 24 month embargo labeled 'U of I Access', the embargo will last until 2024-05-01
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Modelling and correction of retinal pathologies caused by mitochondrial DNA mutations via genetic engineering
… nucleases. The recent emergence of mitochondrial base editors has broadened the scope of mtDNA genetic engineering approaches. However, the potential of programmable nucleases or base editors for modifying retinal mtDNA remains unexplored. This thesis first characterised the retinal …
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Genetic correction of Parkinson’s disease using CRISPR-SKIP
… We have recently demonstrated that CRISPR-Cas9 base editors can be used to induce exon skipping by mutating the conserved “AG” dinucleotide within the splice acceptor site preceding each exon. Here we demonstrate skipping of exon 3 in the SNCA gene in mammalian cells in culture by A>G and C>T …
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Precision Gene Editing for Muscle Diseases
… afflicted individual. Gene editing tools like base editors and prime editors enable us to target and correct the underlying genetic causes of these diseases. Base editors enable precise base pair transition edits, while prime editors offer even more versatility, allowing for base pair …
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Modeling structured biological processes with machine learning
… genetic mutations. Next, I consider a model for base editing, where I decompose a complex prediction problem into simpler subproblems and solve one with an autoregressive sequence-todistribution of sequences model. The models enable designing genome editing strategies with optimized outcomes for …
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Engineering programmable nucleases for in vivo and in vitro applications
… my efforts to developing a diagnostic platform based on Argonaute nucleases, called the SPOT (Scalable and Portable Testing) system. This system combines a rapid, highly sensitive, and accurate assay with a battery-powered portable device for COVID-19 diagnosis. It enables the multiplexed …
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Genetic Engineering as a Tool to Investigate Mitochondrial Gene Expression
… mitochondria, in particular the development of base editing, it is now possible to investigate these processes in more detail than ever before. Using these reverse genetics approaches, this work aims to utilise this new toolkit to probe different aspects of mitochondrial gene expression and …
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Quantitative modelling of CRISPR-Cas editing outcomes
… of gene editing from transgenesis to prime editors. I then review the modelling techniques I use in my projects, before covering the state of predictive modelling for genome engineering. In two main results chapters, I discuss my work on modelling base editor outcomes and the effects of DNA …
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Discovery of novel systems for genome editing
… of biofuels, pharmaceuticals, and other bio-based materials. The increasing global challenges in health and sustainability underscore the necessity of genome editing as a transformative approach to address complex biological and medical questions. The experimental validation of Cas9-based …