Covalent conjugation of oligonucleotides with cell-targeting ligands

被引：15

作者：

Alam, Md. Rowshon ^{[1
,3
]}

Ming, Xin ^{[1
]}

Nakagawa, Osamu ^{[1
,4
]}

Jin, Jian ^{[2
]}

Juliano, R. L. ^{[1
]}

机构：

[1] Univ N Carolina, UNC Eshelman Sch Pharm, Div Mol Pharmaceut, Chapel Hill, NC 27599 USA

[2] Univ N Carolina, UNC Eshelman Sch Pharm, Ctr Integrat Chem Biol & Drug Discovery, Chapel Hill, NC 27599 USA

[3] NITTO DENKO Avecia, Cincinnati, OH 45215 USA

[4] Osaka Univ Pharmaceut Sci, Takatsuki, Osaka 5691094, Japan

来源：

BIOORGANIC & MEDICINAL CHEMISTRY | 2013年 / 21卷 / 20期

关键词：

Oligonucleotide; Conjugation; siRNA; Antisense; Splice switching; Ligand; Receptor; Delivery; IN-VIVO DELIVERY; RECEPTOR-MEDIATED ENDOCYTOSIS; PROTEIN-COUPLED RECEPTORS; ANTISENSE OLIGONUCLEOTIDES; INTRACELLULAR DELIVERY; SIRNA OLIGONUCLEOTIDES; PENETRATING PEPTIDES; LIPOPHILIC SIRNAS; ACID THERAPEUTICS; RNA;

D O I：

10.1016/j.bmc.2013.05.037

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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.

引用

页码：6217 / 6223

页数：7

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