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 14 of 14 for “"AAV vector"”.
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Improving the Safety and Efficacy of AAV Gene Therapy for Hemophilia A
… long-term efficacy after adeno-associated viral (AAV) vector delivery of the F8 gene to hepatocytes. To address questions of safety, we treated privately owned dogs with a liver directed AAV serotype 8 vector encapsidating a canine F8 gene. These dogs were followed long-term for efficacy of the …
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Developing Adeno-Associated Viral Vectors As A Gene Editing Platform
The success 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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Developing an astrocyte-selective AAV-ADAMTS4 gene therapy to promote repair after spinal cord injury
… therapeutic molecules. Adeno-associated viral (AAV) vector gene therapy, in particular, has emerged as the vector of choice for safe, robust and long-term transgene expression in the central nervous system (CNS). Since astrocytes play an important role in SCI pathology, the first objective of …
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LIVER FIBROSIS IMPAIRS HEPATOCYTE TRANSDUCTION BY AAV VECTORS
Adeno-associated viral vectors (AAVs) are the most promising tools for liver directed gene therapy. However, integrity of hepatic architecture has been considered pre-requisite for efficient gene delivery and clinical studies have been addressed toward patients with no or negligible hepatic damage …
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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
… 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 interaction with Ly6a, a …
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Prevention of Duchenne Muscular Dystrophy by CRISPR/Cas Therapeutic Genome Editing
… Cas9 nuclease in conventional single-stranded AAV and CRISPR single guide RNAs in double-stranded self-complementary AAV. This strategy significantly reduces the amount of AAV vector needed for therapeutic genome editing and enhances dystrophin restoration after delivery into a mouse model of …
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Molecular Profiling and Mechanisms of Cerebrovascular Function in Health and Neurodegeneration
… 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 dose of gene therapy …
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Cellular mechanisms of environmental enrichment: Novel discovery-based strategies for target identification for neuropsychiatric disorders
… beta, with a novel adeno-associated viral (AAV) vector in the nucleus accumbens shell (NAcSh) of rats and found increases in depression-like and cocaine taking behaviors. Additionally, GSK3 beta knockdown significantly reduced the activity of tonically active interneurons in the shell. The …
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Development of the Clinical Application of miRNA with Proregenerative Effect on the Heart
… once expressed in the mouse heart using viral vectors, to induce cardiac regeneration after myocardial infarction. Objective: As a first step towards clinical translation, in this study we assess the efficacy of the pro-regenerative miR-199a after myocardial infarction, delivered using an AAV …
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Integrin alpha(v) and Focal adhesion kinase - promising targets to limit smooth muscle cell migration
… eines FAK Inhibitors, der über ein AAV Vektorsystem übertragen wurde, gehemmt werden konnte. So stellt die Blockade der Integrin vermittelten Signalkaskaden ein vielversprechendes Ziel für die Inhibition der Restenose nach PTCA dar. Im ersten Teil der Arbeit konnten wir nach …
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IMPACT OF DRP1 ACTIVATION, UNFOLDED PROTEIN RESPONSE INDUCTION AND FGF21 LEVELS IN DMD PROGRESSION
… excessive fission in mdx mice injecting an AAV-vector encoding for a validated Drp1 shRNA. We observed that the viral genome was still present after 11 weeks from the injection, suggesting a therapeutic action in preserving muscles. Indeed, AAV-shDrp1 had a positive effect on in situ muscle …
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Comparing Differentiated Rod And Cone Transcriptomes Reveals Rax As A Controller Of A Defined Cone Gene Regulatory Program
… We developed an Adeno-associated viral (AAV) vector-based transfer method to isolate rods and cones by fluorescence-based sorting. To isolate “pure” rod and cone populations from the same retinal samples we designed a double expression cassette (“double fluo”) encoding mCherry under the …
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Preclinical evaluation of a novel AAV-GDF5 vector in a 6-OHDA model of Parkinson’s disease and evaluation of the effect of 6-OHDA on the expression of HDACs
… benefits. For this reason, the use of viral vectors to deliver neurotrophic factor genes has become a popular alternative, as this would allow for prolonged expression of the neurotrophic factor. In this study, we used an AAV2/5 vector to deliver GDF5 to the substantia nigra of rats, both at …