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 20 of 34 for “"Adeno-associated virus (AAV)"”.
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Using a Mammalian Virus to Create Plants for Site-Specific Transgene Insertion
… insertion of transgenes using features of the adeno-associated virus (AAV) life cycle. In the absence of helper virus, AAV integrates into a specific location on human chromosome 19, the AAVS1 locus. The sequence for AAV integration was introduced into the model plant Arabidopsis thaliana using …
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Expanding the genome editing toolbox for biomedical applications
… such strategy is CRISPR-READI, which combines adeno-associated virus (AAV)-mediated gene delivery with electroporation of Cas9-RNPs. The AAV delivers transgenes (up to 4.7 kb) as ssDNA to the zygotes, which is followed by electroporation of Cas9-RNPs. This results in improved and precise genome …
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Medical Engineering and Medical Physics: Metagenomic Sequencing for Viral Diagnostics and Discovery
… for the directed evolution of viral vectors. Adeno-associated virus (AAV) is a non-pathogenic virus that infects humans and commonly used as a vector for gene therapy. However, natural AAV serotypes tend to accumulate in the liver, leading to toxic side-effects when higher doses are used to …
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Expression and regulation of genes associated with CNS regeneration and plasticity
… (CI)-induced axon regeneration 3 D. Regeneration associated genes (RAGs) 4 E. Epigenetics 5 F. Neuroepigenetics in CNS injury 8 G. Next generation sequencing (NGS) in epigenomic studies 10 H. Aims of this study 11 II. Materials and methods 13 1. Animals and surgical procedures 13 2. In vivo 5-aza …
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Highly efficient Cas9 mediated transcriptional programming and delivery via Adeno-Associated Virus
… only approved vector for human delivery is the Adeno-Associated Virus (AAV). This convenient virus allows for targeting of various tissue types with high efficiency and little risk of integration or immunogenicity. Unfortunately, the virus is limited to a genomic payload of 4.7kb, which can be …
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OPTIMIZING GENE THERAPY: THE IMPACT OF SMALL MOLECULE MODULATORS ON ADENO-ASSOCIATED VECTOR TRANSDUCTION AND INTEGRATION
Adeno-associated virus (AAV) is a widely used vehicle for gene delivery but a limitation to AAV gene therapy can be inefficient transgene expression from AAV vectors. One approach to improve AAV transduction has been to combine the vector with small molecule modulators that disrupt cellular …
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CHARACTERIZATION OF THE AAV-PHP.B CAPSID AND ITS INTERACTION WITH THE LY6A RECEPTOR: IMPLICATIONS FOR RECEPTOR-TARGETED VECTOR ENGINEERING
… barrier (BBB). Recently, in murine models, the adeno-associated virus (AAV) vector AAV-PHP.B has demonstrated an ability to overcome this barrier and elicit robust transgene expression throughout the CNS when administered intravenously. AAV-PHP.B achieves this through an engineered receptor …
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Generation of focal Depdc5 knockout mouse model and implications for focal epilepsy
… DEPDC5 is the most commonly identified gene associated with familial focal epilepsy and malformations of cortical development. It is also associated with an increased risk of Sudden-Unexplained Death in Epilepsy (SUDEP). It remains unknown whether seizures due to DEPDC5 loss are a result of …
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Investigating intrathecal adeno-associated virus vectors for the study of analgesic interactions at the spinal level
… at the spinal level: develop the use of adeno-associated virus (AAV) vectors for gene-transfer to sensory neurons where the expression of involved proteins can be modulated, and to further characterize these interactions by understanding of the cellular processes underlying their …
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Adeno-Associated Virus-Based Gene Delivery Approaches for Tissue Engineering
… with minimal interference and at low cost. Adeno-associated virus (AAV) is employed as the candidate gene delivery vehicle due to its ability to infect many cell types and good safety profile in humans, which bode well for clinical applications. The body of work is grouped into three parts. …
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Neuroprotektion und Neurorestauration im MPTP Modell der Parkinson Erkrankung
… In der vorliegenden Studie wurde untersucht, ob adeno-associated virus (AAV)-5 Vektoren, injiziert ins Striatum von C57Bl/6-J Mäusen in hoher und niedriger Titerkonzentration, neuronale oder astrozytäre Produktion von GDNF hervorrufend, unterschiedliche Einflüsse auf die protektive und …
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Molecular Profiling and Mechanisms of Cerebrovascular Function in Health and Neurodegeneration
… integrity. Lastly, using a combination of an adeno-associated virus (AAV) approach in combination with a cell type-specific promoter (CLDN5) towards brain endothelial cells, we develop an AAV vector for effective gene therapy delivery to the cerebrovasculature. We demonstrate that a single …
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Expanding the toolkit of protease activatable viruses to improve their versatility and modularity
Adeno-associated virus (AAV) has emerged as a promising gene delivery vector because of its non-pathogenicity, simple structure and genome, and low immunogenicity compared to other viral vectors. However, its adoption as a safe and effective gene therapy treatment for disease relies on targeting …
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Molecular mechanisms of neuronal homoeostasis in vivo
… I show that the expression of Kir2.1 through adeno-associated virus (AAV) does not cause any adverse effects in the dentate gyrus nor the CA1 of the mouse hippocampus. I go on to use slice patch clamp methods to measure the change in electrical properties of granule cells in the dentate gyrus …
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Adeno-Associated Mediated Transduction of Sensory Epithelia Within the Guinea Pig Cochlea
… R. L., M.S., Autumn 2006 Pharmaceutical Sciences Adeno-associated virus mediated transduction of sensory epithelia within the guinea pig cochlea Chairperson: Dr. David J. Poulsen, PhD. Hearing loss is the most common disability of the senses and affects 250 million people worldwide. This condition …
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Adeno-Associated Mediated Transduction of Sensory Epithelia Within the Guinea Pig Cochlea
… R. L., M.S., Autumn 2006 Pharmaceutical Sciences Adeno-associated virus mediated transduction of sensory epithelia within the guinea pig cochlea Chairperson: Dr. David J. Poulsen, PhD. Hearing loss is the most common disability of the senses and affects 250 million people worldwide. This condition …
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Pathogenesis and Therapy of Mitochondrial Diseases
… gene therapy approach for mitochondrial diseases associated with mutations in nuclear-encoded genes. In particular, I test the use of a novel adeno-associated virus (AAV) capsid (PHP.B) as a gene therapy platform to ameliorate the neurological symptoms of a pre-clinical mouse model of …
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Study of the aryl hydrocarbon receptor as a target for rational drug design
… its ligand and DNA binding. In chapter 3, an adeno-associated virus (AAV) expression system was constructed to express an AhR deletion contruct CΔ553 (aa1-295) for tumor injection. Western blot shows the expression of CΔ553 (aa1-295) in hela cells infected by AAV-553, but the low yield of …
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Viral Delivery of the Fat-1 Gene to Treat Post-Traumatic Arthritis with Diet-Induced Obesity
… DMM surgery. In vitro testing of both lentivirus (LV) and adeno-associated virus (AAV) was completed to confirm functionality and conformation of the Fat-1 enzyme after transduction. Male WT mice were placed on an omega-6 rich high-fat diet (60% fat) and underwent DMM surgery; either local …
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Mechanisms of Peripheral Sensitization in Inflammatory Knee Pain
… is to deliver excitatory or inhibitory genes via adeno-associated virus (AAV). However, delivering genes into DRG neurons by injection of AAVs into the peripheral organs has had limited success due to the large distances involved. Here I show that the newly engineered serotype, AAV-PHP.S, can …
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