Covalent conjugation of oligonucleotides with cell-targeting ligands

详细信息    查看全文

• 作者：Md. Rowshon Alam ; Xin Ming ; Osamu Nakagawa ; Jian Jin ; R.L. Juliano
• 关键词：Oligonucleotide ; Conjugation ; siRNA ; Antisense ; Splice switching ; Ligand ; Receptor ; Delivery
• 刊名：Bioorganic and Medicinal Chemistry
• 出版年：2013
• 出版时间：15 October, 2013
• 年：2013
• 卷：21
• 期：20
• 页码：6217-6223
• 全文大小：954 K

文摘

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.