The delivery of therapeutic oligonucleotides

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ID: 305883
2016
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Abstract
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.
Reference Key
openalex_W2336977304 Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Rudolph L. Juliano
Journal Nucleic Acids Research
Year 2016
DOI
10.1093/nar/gkw236
URL
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