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 9 of 9 for “"viral gene therapy"”.
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Quantitative analysis of non-viral gene therapy in primary liver culture systems
Gene therapy has the potential to cure thousands of diseases caused by genetic abnormalities, provide novel combination therapies for cancers and viral infections, and offer a new and effective platform for next generation vaccines. However, after more than three decades of research and development …
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Enhanced polymeric nanoparticles for gene delivery
The potential of gene therapy to treat disease and improve human health is tremendous. The failure of viral gene therapy clinical trials due to toxicity, immunogenicity, and carcinogenicity has been tragic and strongly motivates a non-viral approach. However, non-viral gene delivery is currently …
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Production of AAV vectors for gene therapy : a cost-effectiveness and risk assessment
Gene therapy is a promising modality for the potential treatment of rare Mendelian diseases. To date a number of high profile proof-of-concept studies within the industry have demonstrated the significant disease-correcting promise of this therapeutic strategy. One of the major hurdles that remains …
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Baculovirus as a gene delivery and expression vector in mammalian cells
… free radical formation and organ failure. Viral gene therapy, such as BacMam gene transfer technology (Baculovirus-based expression in mammalian cells) is an attractive option for reducing I/R injury due to its high safety profile compared to mammalian viruses. However, limiting factors, …
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Photo-responsive degradable poly (beta-amino ester) toward non-viral gene delivery
… ester) (PBAE) is one of ideal candidates for non-viral gene therapy due to its desired gene delivery efficiency, biocompatibility, and biodegradability. In order to further improve the gene delivery efficiency of PBAE by facilitating the intracellular DNA dissociation, we herein report the design …
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Quantitative analysis of cytokine-induced hepatocellular death in the context of hepatotoxic therapeutics
Numerous therapeutics, such as viral gene therapy vectors, have unintended toxicity in part due to interactions with inflammatory cytokine signaling to elicit hepatocyte death, thus limiting their clinical use. Although much is known about how cytokines and certain therapeutics individually induce …
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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
… 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 neurodegenerative disorder caused by an unstable expansion of a single …
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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
… 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 neurodegenerative disorder caused by an unstable expansion of a single …
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The development of novel methods for the targeting and manipulation of neural circuits in vivo
… key component of several psychiatric and neurodegenerative disorders such as Alzheimer’s disease or schizophrenia. However, our ability to precisely and safely manipulate neural networks for research and deliver network-specific therapy remains limited. To address this problem, our lab recently …