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 13 of 13 for “"antisense oligonucleotides (ASOs)"”.
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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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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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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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Development and Application of a Novel Assay for Rapid Identification of Cellular Prion Protein Modulators
… to reduce cellular prion protein (PrPC) levels. Antisense oligonucleotides (ASOs) currently represent the only therapeutic strategy in clinical trials, showing promising potential, but further investigations are needed to assess their long-term efficacy. In parallel, the search for novels, …
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Antisense Reduction of the Protein Tau Attenuates Neuronal Hyperexcitability and Permits Clearance of Intraneuronal Tau Accumulations in vivo
… sought to directly target the protein tau using Antisense Oligonucleotides (ASOs) to reduce total expression of tau <italic> in vivo </italic> and assess if such a reduction could be therapeutically beneficial. To first test the feasibility of reducing tau in the adult animal, we identified ASOs …
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Interrogating Dux4 Mrna 3′ End Processing
… Dux4 mRNA 3′end formation, we designed antisense oligonucleotides (ASOs) to impair the production of polyadenylated Dux4. Prior to antagonizing Dux4 CPA, we demonstrate, in proof of principle experiments that ASOs directed toward required CPA regulatory elements can impair gene …
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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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BETTING ON ALTERNATIVES: TARGETING TELOMERIC DILNCRNA AND BET PROTEINS IN ALT CANCER
… In fact, inhibition of dilncRNA functions using antisense oligonucleotides (ASOs) dampens DDR in a sequence-specific matter, impeding repair. Consistent with the persistence of DDR activation at ALT telomeres, we observed increased transcription of telomeric dilncRNA in ALT cells compared to …
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RNA Knockdown of the Immune Checkpoint VISTA Promotes Tumor Regression
… cell surface due to endosomal recycling, we used Antisense oligonucleotides (ASOs) to knockdown VISTA expression on myeloid cells. ASOs are synthetically generated single stranded oligonucleotides that alter messenger RNA to either diminish, recover, or adapt protein expression. We hypothesize …
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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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RNA-MEDIATED PHASE SEPARATION OF 53BP1: CONNECTING CONDENSATE BIOPHYSICS TO DNA DAMAGE RESPONSE
… relevance in DNA repair. Finally, we prove that antisense oligonucleotides (ASOs) targeting telomeric transcripts reduce protein-RNA binding affinity, providing a possible mechanistic explanation for ASO-induced dissolution of DDR foci. Collectively, we propose that the GAR motif of 53BP1 drives …
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DESIGN AND DEVELOPMENT OF RNA-BASED DRUGS USING MOLECULAR DYNAMICS SIMULATIONS AND ARTIFICIAL INTELLIGENCE METHODS
… interfering RNAs (siRNAs), microRNAs (miRNAs), antisense oligonucleotides (ASOs), circular RNAs (circRNAs), and aptamers. Despite their significant potential, these therapeutics face important limitations, including susceptibility to RNase degradation, high net negative charge, and rapid renal …
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Characterization of ARV1-Mediated Sterol Transport in Yeast and Mammalian Systems
… Arv1p function. In mammalian cells we utilized antisense oligonucleotides (ASOs) to decrease 𝘈𝘙𝘝1 expression 𝘪𝘯 𝘷𝘪𝘵𝘳𝘰 and 𝘪𝘯 𝘷𝘪𝘷𝘰. In the yeast model, loss of Arv1p function results in sensitivity to modulators of sphingolipid homeostasis and aberrant accumulation of exogenous sterols. …