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 20 of 179 for “"gene-editing"”.
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Gene editing in Aedes aegypti
… to control these diseases, developing novel, genetics-based, control strategies to limit the transmission of disease is urgently needed. Increased knowledge about mosquito-pathogen relationships and the molecular biology of mosquitoes now makes it possible to generate transgenic mosquito …
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Precision Gene Editing for Muscle Diseases
… to do the many incredible things. Consequently, genetic mutations that cause muscle disease can have a profound effect on the 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 …
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Development and Characterization of Viral-Based Gene Editing In Vivo
… can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV vectors are a preferred delivery vehicle for tissue-directed gene therapy because of their ability to achieve sustained expression from largely non-integrating episomal genomes. However, for genome …
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Developing Adeno-Associated Viral Vectors As A Gene Editing Platform
… of adeno-associated viral (AAV) vectors for gene replacement applications has spurred interest to repurpose AAV into a platform for CRISPR/Cas9 gene editing. Yet, AAV vector behaviors that are critical for gene editing have been unexplored. To safely and effectively combine AAV and …
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Investigating the role of chromatin modifications in CRISPR/Cas9 gene editing
… have been shown to impede CRISPR/Cas9 editing efficiency. Conversely, Cas9 can open previously inaccessible regions of DNA, and transcriptionally silent targets can usually be edited without difficulty. In order to address this paradox, we have developed a method that exploits the …
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Probing single-molecule dynamics of molecular motors and gene editing proteins
… two proteins systems: Molecular Motors and Gene Editing Proteins. Kinesin walks on cellular roads called microtubules and transports cellular cargos. Kinesin is one of the many types of molecular motors present in living cells. Generally, multiple kinesin motors are present on a single cargo …
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CPISPR-CAS: From a Prokaryotic Immune System to a Gene Editing Tool
… Repeats (CRISPR) and their associated genes (cas) encode an adaptive, small-RNA-based immune system that protects prokaryotes from infectious phages and plasmids. CRISPR-Cas systems can be classified into three types based on their cas gene content. My thesis work focused on two parts. …
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CRISPR/CAS9 mediated gene editing for the improvement of beef and dairy cattle
… on favorable traits, many of which are due to genetic variation. Single nucleotide polymorphisms are responsible for a great deal of either beneficial or undesirable mutations. In this study, we examine how we can use the CRISPR/Cas9 system to induce single-base pair substitutions, thereby …
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Re-engineering of Dendrimer-Based Lipid Nanoparticles for Efficient and Precise HDR-Mediated Gene Editing
CRISPR/Cas gene editing is poised to transform the treatment of genetic diseases. However, limited progress has been made toward precise editing of DNA via Homology Directed Repair (HDR) that requires careful orchestration of complex steps. Rather, many reports of in vivo gene editing rely on an …
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Developing a new efficient CRISPR-Cas9 base Homology Directed Repair oriented gene editing plasmid for Streptomyces
The CRISPR-Cas9 editing mechanism revolutionized genetic engineering, providing a precise and versatile tool for editing the genomes of virtually any organism. Streptomyces are soil dwelling bacteria with a complex life cycle and a reputation for producing a wide array of bioactive compounds, …
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Modeling Alzheimer's Disease Using CRISPR/CAS9 Gene Editing and Induced Pluripotent Stem Cells Reveals Conserved Cellular Mechanisms
… autosomal dominant familial AD (fAD) result from genetic mutations in three key genes: amyloid precursor protein (APP), and two APP processing-related genes (presenilin-1 (PSEN1), and presenilin-2 (PSEN2)), supporting the theory that altered APP metabolism is a central cause of AD. However, which …
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CRISPR gene editing for Cystic Fibrosis: targeting the G542X mutation with base editor engineered virus-like particles
Cystic Fibrosis (CF) is a severe genetic disorder affecting hundreds of thousands of individuals worldwide. Approximately 10% of CF patients currently lack treatment options beyond symptomatic care and face a reduced life expectancy. CRISPR-based gene editing offers the potential for a cure, by …
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Cystic fibrosis gene repair: correction of ΔF508 using ZFN and CRISPR/Cas9 guide RNA gene editing tools
… transmembrane conductance regulator (CFTR) gene with the ΔF508 mutation accounting for approximately 70% of all CF cases worldwide. This thesis investigates whether existing zinc finger nucleases designed in this lab and CRISPR/gRNAs designed in this thesis can mediate efficient …
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Interrogating novel functions of the I kappa B kinases via CRISPR-Cas9 gene editing and small molecule inhibition
… of NF-κB transcription factor activity. Seminal genetic studies in knockout (KO) mouse embryonic fibroblasts (MEFs) have defined two pathways of NF-κB activation; a canonical pathway, activated in response to cytokines such as TNFα/IL-1β, that requires NEMO and predominantly IKKβ catalytic …
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The Design, Synthesis, and Evaluation of Zwitterionic and Cationic Lipids for In Vivo RNA Delivery and Non-Viral CRISPR/Cas Gene Editing
… clinical development for the treatment of many genetic diseases. The use of RNA interference (RNAi) as a therapeutic is an exciting and rapidly developing field that offers a promising alternative to small molecule drugs for the treatment of dysregulatory diseases, including cancer. Small …
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Trade implications of gene-edited wheat under different regulatory scenarios
… a challenge. New breeding techniques (NBTs) or gene editing are rapidly emerging as alternative and sustainable methods of improving methods wheat traits. Although these innovative breeding methods contribute to higher yields and develop crops with valuable traits, the regulatory status and the …
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The impact of the national bioengineered food disclosure standard and its methods of disclosure on consumer preferences for gene edited and genetically modified foods
… food produced with, or containing, certain plant genetic improvement techniques - such as genetic modification. The standard introduces the new term “Bioengineered” on the approved label, which can be disclosed through methods such as a symbol, text, QR code, and mobile text message. Advances in …
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Elucidating the molecular mechanisms of p110δ activation in T cell antigen receptor signalling
… used a combination of proteomic, biochemical and gene editing approaches to identify proteins that are involved in the regulation of p110δ activity during TCR signalling. This work has optimised the AviTag affinity purification system for the isolation of endogenous p110 isoforms from primary …
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Development and use of a synthetic biology toolkit to enhance high value lipid production in Nannochloropsis oculata
… quantity of EPA to be commercially viable. Gene editing has shown significant potential to address this issue though cellular and specifically lipid metabolic engineering; however, the use of established CRIPSR systems is prohibitively expensive. This MRes thesis aimed, therefore, to develop …
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