Journal
NUCLEIC ACIDS RESEARCH
Volume 36, Issue 12, Pages 4158-4171Publisher
OXFORD UNIV PRESS
DOI: 10.1093/nar/gkn342
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Funding
- NIGMS NIH HHS [P01 GM059299] Funding Source: Medline
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The potential use of antisense and siRNA oligonucleotides as therapeutic agents has elicited a great deal of interest. However, a major issue for oligonucleotide-based therapeutics involves effective intracellular delivery of the active molecules. In this Survey and Summary, we review recent reports on delivery strategies, including conjugates of oligonucleotides with various ligands, as well as use of nanocarrier approaches. These are discussed in the context of intracellular trafficking pathways and issues regarding in vivo biodistribution of molecules and nanoparticles. Molecular-sized chemical conjugates and supramolecular nanocarriers each display advantages and disadvantages in terms of effective and nontoxic delivery. Thus, choice of an optimal delivery modality will likely depend on the therapeutic context.
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