The delivery of therapeutic oligonucleotides
| Autor: | Rudolph L. Juliano |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
Drug Oligonucleotide media_common.quotation_subject Gene Transfer Techniques Oligonucleotides Late stage Receptors Cell Surface Context (language use) Computational biology Biology Ligands Small molecule 03 medical and health sciences 030104 developmental biology Nucleic Acids Splice switching Genetics Animals Humans Survey and Summary Intracellular media_common |
| Zdroj: | Nucleic Acids Research |
| ISSN: | 1362-4962 0305-1048 |
| DOI: | 10.1093/nar/gkw236 |
| Popis: | The oligonucleotide therapeutics field has seen remarkable progress over the last few years with the approval of the first antisense drug and with promising developments in late stage clinical trials using siRNA or splice switching oligonucleotides. However, effective delivery of oligonucleotides to their intracellular sites of action remains a major issue. This review will describe the biological basis of oligonucleotide delivery including the nature of various tissue barriers and the mechanisms of cellular uptake and intracellular trafficking of oligonucleotides. It will then examine a variety of current approaches for enhancing the delivery of oligonucleotides. This includes molecular scale targeted ligand-oligonucleotide conjugates, lipid- and polymer-based nanoparticles, antibody conjugates and small molecules that improve oligonucleotide delivery. The merits and liabilities of these approaches will be discussed in the context of the underlying basic biology. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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