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 11 of 11 for “"Adeno Associated Viruses"”.
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Reconstructing Cross-Species Ancestral Adeno-Associated Viruses for Enhanced Gene Therapy Delivery
Adeno-associated viruses (AAV) are one of the most promising vectors for gene therapy because of their established safety, low immunogenicity, and capability to achieve sustained gene expression. However, many naturally occurring AAV variants have limitations in their potency, particularly in …
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Targeted Gene Therapy Approaches for Huntington's Disease: Advancing Treatment through Bioengineered Self-Assembling Peptide Hydrogels and BDNF-Encoded Adeno-Associated Viruses, Applied In Vitro and In Vivo
… accessibility and cost. This phenomenon is associated with progressive age-related pathologies such as neurodegenerative disorders. Also, CNS incapacity to regenerate is an obstacle to efficiently treating neurodegenerative diseases. Huntington's disease (HD) is recognized as a fatal …
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Targeted Gene Therapy Approaches for Huntington's Disease: Advancing Treatment through Bioengineered Self-Assembling Peptide Hydrogels and BDNF-Encoded Adeno-Associated Viruses, Applied In Vitro and In Vivo
… accessibility and cost. This phenomenon is associated with progressive age-related pathologies such as neurodegenerative disorders. Also, CNS incapacity to regenerate is an obstacle to efficiently treating neurodegenerative diseases. Huntington's disease (HD) is recognized as a fatal …
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THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS
… growth and strengthening. Moreover, the use of adeno-associated viruses ensures the efficient, specific delivery of the transgenes to the muscle that requires it. Furthermore, the alternatively spliced variant of Follistatin used in the therapy ensures the prevention of any off-target effects …
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The Role of Fractalkine-mediated Neuroprotection in the Diabetic Retina
… inflammation using recombinant adeno-associated viruses (rAAVs), address the contribution of full-length FKN and ctFKN to retinal inflammation in our models of diabetes. Here, we show that prophylactic administration of sFKN, but not mFKN, prevented vascular and neuronal damage, …
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Engineered Replicases, Synthetic DNA, and Long-Read Sequencing Quality Control for Adeno-Associated Virus Production
… can be treated with modified (recombinant) viruses that deliver therapeutic transgenes. Recombinant adeno-associated viruses (rAAV) are the most promising candidates for treatment, as clinical trials have demonstrated the safety of the vector at low and medium-dose, and three AAV-based …
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Towards the establishment of a highly effective adeno-associated virus production by leveraging next generation sequencing and transcriptomics in HEK293 cells
… the available delivery methods, recombinant adeno-associated viruses (rAAV) have emerged as one of the most promising vectors due to their low immunogenicity, long-term expression even in differentiated cells, and very low rate of integration into the host genome. However, process yields …
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Understanding The Visual And Auditory Defect In Ush2a Mouse Model
… due to limited payload capacity of commonly used Adeno-associated viruses (AAV) ( <4.7 kbp.) To understand the mechanism underlying hearing and visual impairments, a knock in (KI) mouse model (Ush2adelG/delG) was developed using one of the most prevalent human mutations of USH2A, 2299delG. While …
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Analysis, Optimization and Application of AAV Capsid Assembly using Escherichia coli
Recombinant adeno-associated viruses (rAAV) are leading drugs in gene therapy with three products approved by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA). Currently, rAAV vectors are evaluated in many ongoing clinical trials for treating rare genetic diseases and …
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Stimulating angiogenesis into biomaterials through the delivery of growth factors
… for human VEGF15s was cloned into the genome of adeno associated viruses (AAV), which served as a vector for gene transduction of autologous wound healing cells in vivo using the "Gene Activated Matrix" approach. Genetically modified matrix embedded AAV-VEGF155 was loaded into porous PU and …
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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 …