title: Current progress on aptamer-targeted oligonucleotide therapeutics.
abstract: Exploiting the power of the RNAi pathway through the use of therapeutic siRNA drugs has remarkable potential for treating a vast array of human disease conditions. However, difficulties in delivery of these and similar nucleic acid-based pharmacological agents to appropriate organs or tissues, remains a major impediment to their broad clinical application. Synthetic nucleic acid ligands (aptamers) have emerged as effective delivery vehicles for therapeutic oligonucleotides, including siRNAs. In this review, we summarize recent attractive developments in creatively employing cell-internalizing aptamers to deliver therapeutic oligonucleotides (e.g., siRNAs, miRNAs, anti-miRs and antisense oligos) to target cells. We also discuss advancements in aptamer-siRNA chimera technology, as well as, aptamer-functionalized nanoparticles for siRNA delivery. In addition, the challenges and future prospects of aptamer-targeted oligonucleotide drugs for clinical translation are further highlighted.
authors.affiliation: Department of Internal Medicine, University of Iowa, 375 Newton Rd, 5202 MERF, Iowa City, IA 52242, USA.
authors.forename: Justin P
authors.initials: JP
authors.lastname: Dassie
authors.forename: Paloma H
authors.initials: PH
authors.lastname: Giangrande
mesh_terms: D000818:Animals; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000627:therapeutic use; D052157:Aptamers, Nucleotide / Q000008:administration & dosage* / Q000737:chemistry / Q000627:therapeutic use*; D016503:Drug Delivery Systems; D015658:HIV Infections / Q000188:drug therapy; D006801:Humans; D008958:Models, Molecular; D053758:Nanoparticles; D009369:Neoplasms / Q000188:drug therapy; D009841:Oligonucleotides / Q000008:administration & dosage* / Q000627:therapeutic use*; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D052157:Aptamers, Nucleotide; D009841:Oligonucleotides; D034741:RNA, Small Interfering