Sökning: "antisense oligonucleotide"
Visar resultat 1 - 5 av 23 avhandlingar innehållade orden antisense oligonucleotide.
1. Specificity of antisense oligonucleotide derivatives and cellular delivery by cell-penetrating peptides
Sammanfattning : 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. LÄS MER
2. Quantitative NanoSIMS provides subcellular concentration and distribution of oligonucleotide therapeutics
Sammanfattning : 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. LÄS MER
3. Conformationally Constrained Nucleosides : Design, Synthesis, and Biochemical Evaluation of Modified Antisense Oligonucleotides
Sammanfattning : 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. LÄS MER
4. Conformationally Constrained Oligonucleotides for RNA Targeting
Sammanfattning : 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. LÄS MER
5. Characterization of parameters influencing intracellular bioavailability and prediction of intracellular drug exposure
Sammanfattning : 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. LÄS MER