Search for dissertations about: "antisense oligonucleotide"

Showing result 1 - 5 of 23 swedish dissertations containing the words antisense oligonucleotide.

2. 1. Specificity of antisense oligonucleotide derivatives and cellular delivery by cell-penetrating peptides

   Author : Peter Guterstam; Ülo Langel; Matthew Wood; Stockholms universitet; []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; cell-penetrating peptide; splice-switching oligonucleotide; oligonucleotide derivative; Neurochemistry; Neurokemi; Neurochemistry and Molecular Neurobiology; neurokemi med molekylär neurobiologi;

   Abstract : Atypical gene expression has a major influence on the disease profile of several severe human disorders. Oligonucleotide (ON) based therapeutics has opened an avenue for compensating deviant protein expression by acting on biologically important nucleic acids, mainly RNAs. READ MORE

4. 2. Quantitative NanoSIMS provides subcellular concentration and distribution of oligonucleotide therapeutics

   Author : Cécile Becquart; Göteborgs universitet; []
   Keywords : NATURVETENSKAP; NATURAL SCIENCES; NanoSIMS; Antisense Oligonucleotide; Intracellular concentration;

   Abstract : Antisense oligonucleotides (ASOs) represent a powerful therapeutic modality that can selectively modulate gene expression. However, ASOs face two major hurdles that restrict their use in the clinic. The first issue is delivery of the ASO to a tissue of therapeutic interest while reducing exposure to unrelated tissues. READ MORE

5. 3. Conformationally Constrained Nucleosides : Design, Synthesis, and Biochemical Evaluation of Modified Antisense Oligonucleotides

   Author : Oommen P. Varghese; Lars Engman; Jacek Stawinski; Uppsala universitet; []
   Keywords : NATURVETENSKAP; NATURAL SCIENCES; Organic chemistry; chemically modified oligonucleotides; azetidine; aza-ENA-T; cyanomethylene locked; free energy of activation; diaxial interactions; chair conformation; stable duplex; human serum; snake venom phosphodiesterase; antisense agents; RNase H; Organisk kemi; Chemistry; Kemi;

   Abstract : This thesis is concerned with synthesis, structure and biochemical analysis of chemically modified oligonucleotides with potential therapeutic applications. The three types of chemical modifications described here are: (a) A North-East locked 1',2'-azetidine nucleoside (b) A North locked 2',4'-cyanomethylene bridged nucleoside and (c) A 2',4'-aza-ENA-T nucleoside. READ MORE

6. 4. Conformationally Constrained Oligonucleotides for RNA Targeting

   Author : Qing Li; Jyoti Chattopadhyaya; Li-He Zhang; Uppsala universitet; []
   Keywords : NATURVETENSKAP; NATURAL SCIENCES; NATURVETENSKAP; NATURAL SCIENCES; conformationally constrained nucleoside; antisense oligonucleotide; RNA affinity; nuclease stability; RNase H; RNA degradation; Chemistry with specialization in Bioorganic Chemistry; Kemi med inriktning mot bioorganisk kemi;

   Abstract : A short oligonucleotide sequence as in a single-stranded antisense oligo nucleotides (AON) or in double-stranded small interfering RNAs (siRNA) can modulate the gene expression by targeting against the cellular mRNA, which can be potentially exploited for therapeutic purposes in the treatment of different diseases. In order to improve the efficacy of oligonucleotide-based drugs, the problem of target affinity, nuclease stability and delivery needs to be addressed. READ MORE

7. 5. Characterization of parameters influencing intracellular bioavailability and prediction of intracellular drug exposure

   Author : Andrea Treyer; Per Artursson; Gert Fricker; Uppsala universitet; []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; intracellular drug bioavailability; unbound drug concentration; drug disposition; ADME; drug transport; drug metabolism membrane partitioning; phospholipid; drug-drug interaction; antisense oligonucleotide; cell-penetrating peptide; Farmaceutisk vetenskap; Pharmaceutical Science;

   Abstract : This thesis work investigates factors influencing intracellular drug disposition. An experimental method for measurement of intracellular bioavailability (Fic), was used throughout. Fic is defined as the ratio between the unbound drug concentration inside the cell and the compound concentration in the cell exterior. READ MORE