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 8 of 8 for “"Adeno-associated viral vectors"”.
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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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On the Rational Design of Adeno-Associated Viral Vectors as Precision Tools in Neuroscience
Contains fulltext : 309684.pdf (Publisher’s version ) (Open Access)
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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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Flow cytometry quantitation of dopamine receptor D2 loss as a sensitive measure of Huntington's Disease progression in mouse neurons
… genetically by delivering transgenes via adeno associated viral vectors, and that a small molecule with only subtle transcriptional effects (cystamine) fails to rescue D2GFP loss. I hope that this system can be of great utility in the validation of effective therapeutic interventions for …
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Molecular therapy for mucopolysaccharidosis Type I
… of intracerebroventricular (ICV) infusion of adeno-associated viral vectors in order to mediate gene transfer and expression of IDUA in the brain. Infusion of an AAV serotype 8 vector into neonatal MPS I mice resulted in widespread long-term expression of high levels of IDUA throughout the …
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Activation of 5-HT1A Receptors in the Nucleus Tractus Solitarius Facilitate Ventilatory Responses to Hypoxic Hypercapnia and Promote Sympathetic Recovery Following Hypotensive Hemorrhage
… facilitate chemoreflex responses. We utilized viral-mediated knockdown of the rat 5-HT<sub>1A</sub> receptor in the caudal NTS (cNTS) to examine the role of 5-HT<sub>1A</sub> receptors on compensation following hemorrhage and ventilatory responses to hypoxic/hypercapnia. Sequences encoding a …
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Optogenetic dissection of the dopaminergic circuitry involved in memory consolidation
… knock-in mice and a Cre-dependent adeno-associated viral vectors, CAergic neurons in VTA and LC were selectively tagged with channelrhodopsin-2 (ChR2). Conditional ChR2 expression made it possible to reliably identify CAergic neurons during unit recording sessions in freely moving …
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Gene therapy for axon regeneration
… or acquired condition. In this dissertation, adeno-associated viral (AAV) vectors were used to deliver transgenes into cortical neurons and the corticospinal tract, because regeneration of this neuronal pathway is a key event to restore motor function. One of the advantages of gene therapy is …