期刊
BIOORGANIC & MEDICINAL CHEMISTRY
卷 21, 期 20, 页码 6217-6223出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.bmc.2013.05.037
关键词
Oligonucleotide; Conjugation; siRNA; Antisense; Splice switching; Ligand; Receptor; Delivery
资金
- NCI NIH HHS [R01 CA151964] Funding Source: Medline
A continuing problem in the area of oligonucleotide-based therapeutics is the poor access of these molecules to their sites of action in the nucleus or cytosol. A number of approaches to this problem have emerged. One of the most interesting is the use of ligand-oligonucleotide conjugates to promote receptor mediated cell uptake and delivery. Here we provide an overview of recent developments regarding targeted conjugates, including use of peptides, carbohydrates and small molecules as ligands. Additionally we discuss our own experience with this approach and point out both advantages and limitations. (C) 2013 Elsevier Ltd. All rights reserved.
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