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 46 for “"antisense oligonucleotides"”.
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Selective targeting of MYC by antisense oligonucleotides
… MYC. The goals of this research were to develop antisense oligonucleotides specifically targeted against the MYC mRNA to achieve potent inhibition of MYC translation, and to characterize the activity of these molecules as specific modulators of MYC expression and as prototypical MYC-directed …
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Improving Cardiac Delivery of Antisense Oligonucleotides with Peptidomimetic Targeting Agents
… strategy. Here, we investigate the delivery of antisense oligonucleotides (ASOs) to inhibit the expression of an overexpressed miRNA in CVD, miRNA-21. One of the challenges in delivering ASOs and other gene therapies is achieving delivery to the desired tissue before the therapeutic is …
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Peptide-mediated delivery of antisense oligonucleotides and chemotherapeutics across biological barriers
… methods for the peptide-mediated delivery of antisense oligonucleotides (ASOs) and chemotherapeutics. First, we address the issue that the optimal peptide sequence for the delivery of a macromolecular cargo is often context-dependent and specific to that cargo. With one class of ASO, we …
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SPINAL MUSCULAR ATROPHY ORGANOIDS REVEAL DEVELOPMENTAL DEFECTS RESCUED BY ANTISENSE OLIGONUCLEOTIDES TREATMENT
… hyperexcitability. Optimized peptide-conjugated antisense oligonucleotide tested in SMA SCOs successfully reverted both morphological and functional deficits. Of note, SMA cerebral organoids displayed similar functional deficits, revealing the widespread impact of the disease, beyond the spinal …
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Developing in vitro models to elucidate the mechanisms of toxicity of locked nucleic acid gapmer antisense oligonucleotides.
Antisense oligonucleotides are an emerging class of RNA therapeutics that have recently received several approvals from the clinic. Antisense oligonucleotides act at the RNA level to either degrade target RNA, or to regulate splicing of a particular gene, and have been applied to treat a variety of …
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Design, synthesis, and analysis of conjugated activators for modulation of alternative splicing
… and valproic acid, to viral vectors encoding antisense oligonucleotides. In particular, a synthetic peptide-oligonucleotide conjugate containing a short chain of arginine-serine dipeptides to mimic the RS domain has been shown to redirect splicing patterns. However, little work has been done …
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Thermodynamics and kinetics of antisense oligonucleotide hybridization to a structured mRNA target
Antisense oligonucleotides have the potential to selectively inhibit the expression of any gene with a known sequence. Antisense-based therapies are under development for the treatment of infectious diseases as well as complex genetic disorders. Although there have been some remarkable successes, …
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Role of protein kinase C isoforms in human breast tumor cell survival
… MCF-7 and MDA-MB-231 human breast tumor cells by antisense oligonucleotides significantly impaired survival following radiation insult. Antisense oligonucleotides were shown to be effective and selective inhibitors of specific PKC isoforms. The PKC delta inhibitor, rottlerin reduced cell survival …
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Bioconjugate Strategies for Antisense Therapeutic Delivery to Glioblastoma Stem Cells
Antisense therapeutics, including antisense oligonucleotides (AONs) and small interfering ribonucleic acids (siRNAs), are powerful tools for regulating genes, making them a promising therapy for diseases such as cancer where oncogenic genes are over-expressed. The delivery of antisense therapeutics …
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ENDOGENOUS ANTIVIRAL PROPERTIES OF RED BLOOD CELL-DERIVED EXTRACELLULAR VESICLES AND THEIR AUGMENTATION OF ANTIVIRAL OLIGONUCLEOTIDE THERAPY
… and their use as delivery vehicles for antisense oligonucleotides (ASOs). RBCEVs inhibited both pseudotyped and authentic SARS-CoV-2 infections in a dose-dependent manner by blocking viral entry. ASOs targeting conserved viral genes (helicase, 3CLPro, PLpro, RdRp, and TRS), delivered via …
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Development of Oligonucleotide-directed proximity-interactome MAPping (O-MAP), for characterizing RNA-protein interactions and higher order subnuclear architecture in situ
… or enriching for interacting proteins using antisense oligonucleotides of the target RNA bound to a resin. These approaches are either impossible for smaller structures or are plagued with nonspecific binding and low specificity after exposure to crude lysate. To address these problems, …
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Systematic Interrogation of the Stiffness-sensitive Transcriptome in Mesenchymal Stromal Cells Reveals an Immunomodulatory LncRNA CYTOR
… in gene abundance. Knockdown of CYTOR using antisense oligonucleotides enhances the expression of numerous mechanoresponsive cytokines and chemokines to levels exceeding what is achievable by modulating matrix stiffness alone. Taken together, these findings reveal previously unexplored …
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Insights from biomolecular condensates into disease and drug development
… this condensate partitioning work to show that antisense oligonucleotides (ASOs), nucleic acid-based therapeutics targeting RNA, partition into and modulate certain condensates, and that specific chemical modifications can alter this partitioning behavior. Ultimately, by considering the …
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Paxillin is a Novel Regulator of Xenopus Oocyte Maturation
… Paxillin expression using RNA interference and antisense oligonucleotides completely abrogates steroid-triggered meiotic resumption. Detailed signaling studies reveal that Paxillin is acting early in the kinase cascade, as it is required for accumulation of MOS protein and complete activation of …
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Synthesis and bio-pharmacological activity of antisense phosphatidyl-oligonucleotides
Lipid conjugated oligonucleotides are of great interest in the field of antisense oligonucleotides used for functional genomics, gene target validation and therapeutic pourpose. Although various lipid conjugates of oligonucleotides have already been prepared, it was not possible until now to …
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Glycine receptors in the developing rat spinal cord
… (brPTB). Treatment of neurons in culture with antisense oligonucleotides to "knock down" one of the Nova-1 variants altered the expression of GlyR α2N. These results suggest that the relative levels of the variants of Nova-1 and brPTB may play a role in the developmental regulation of GlyR α2N. …
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Achieving Cell-Specific Delivery of Multiple Oligonucleotide Therapeutics with Aptamer Chimeras
… aptamer delivered splice–switching oligonucleotides, a form of antisense technology, improving their efficacy, and potentially increasing their therapeutic viability. The ability to deliver antisense oligonucleotides to the nuclei of cancer cells has the potential for other …
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Pharmacological regulation of c-myc gene expression in human breast cancer cells
… Furthermore, MCF-7 cells treated with c-myc antisense oligonucleotides exhibited cytoplasmic lipid droplets, similarly to the quinidine-treated cells, suggesting that suppression of Myc may play a causative role in the induction of more differentiated phenotype by quinidine in human breast …
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MicroRNAs in ALS: Defining Cell-Type Specific Expression, Developing Methods to Modulate MicroRNAs in vivo, and Identifying Novel Therapeutic Targets
… improve survival or disease duration. Therefore, antisense oligonucleotides may be used to successfully inhibit miRNAs throughout the brain and spinal cord, and miR-155 is a promising new therapeutic target for human ALS.</p>
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Investigating and Reprogramming RNA Folding with Molecular Probes
… RNA sequences are reprogrammed by short antisense oligonucleotides (ASOs) to fold into six different 3D wireframe polyhedra. How each type of structural feature in the polyhedra affects the stability of local base pairs is revealed using dimethyl sulfate mutational profiling with …
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