title: Covalent conjugation of oligonucleotides with cell-targeting ligands.
abstract: 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.
authors.affiliation: Division of Molecular Pharmaceutics, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, United States; NITTO DENKO Avecia, 8560 Reading Road, Cincinnati, OH 45215, United States.
authors.forename: Md Rowshon
authors.initials: MR
authors.lastname: Alam
authors.forename: Xin
authors.initials: X
authors.lastname: Ming
authors.forename: Osamu
authors.initials: O
authors.lastname: Nakagawa
authors.forename: Jian
authors.initials: J
authors.lastname: Jin
authors.forename: R L
authors.initials: RL
authors.lastname: Juliano
mesh_terms: D002467:Cell Nucleus / Q000737:chemistry; D006801:Humans; D008024:Ligands; D009841:Oligonucleotides / Q000138:chemical synthesis / Q000737:chemistry*; D034741:RNA, Small Interfering / Q000737:chemistry*
chemical_list: D008024:Ligands; D009841:Oligonucleotides; D034741:RNA, Small Interfering
keywords: Antisense; Conjugation; Delivery; Ligand; Oligonucleotide; Receptor; Splice switching; siRNA