title: Sequence-defined polymers for the delivery of oligonucleotides.
abstract: Short synthetic oligonucleotides (ONs) are a group of therapeutic molecules with enormous clinical potential owing to their high specificity and ability to target the expression of virtually any single or group of genes. Clinical translation of ONs is hampered by the inadequate bioavailability in the target cells due to the substantial extracellular and intracellular barriers exposed to these molecules. Different cationic polymers have been successfully deployed for the delivery of ONs. However, heterogeneous nature of these classical polymers is not suitable for clinical applications and hence vectors with completely defined structure are required. In this review, we discuss recent advances with sequence-defined polymers and their application for the delivery of short ONs.
authors.affiliation: Pharmaceutical Biotechnology, Department of Pharmacy and Center for Nanoscience (CeNS), Ludwig-Maximilians-University, Munich, Germany.
authors.forename: Taavi
authors.initials: T
authors.lastname: Lehto
authors.forename: Ernst
authors.initials: E
authors.lastname: Wagner
mesh_terms: D000595:Amino Acid Sequence; D000818:Animals; D018014:Gene Transfer Techniques; D006801:Humans; D053758:Nanoparticles / Q000737:chemistry*; D009841:Oligonucleotides / Q000008:administration & dosage*; D010455:Peptides / Q000737:chemistry*; D011073:Polyamines / Q000737:chemistry*; D034741:RNA, Small Interfering / Q000008:administration & dosage*
chemical_list: D009841:Oligonucleotides; D010455:Peptides; C531249:Poly(amidoamine); D011073:Polyamines; D034741:RNA, Small Interfering
keywords: cell-penetrating peptides; nonviral delivery; oligonucleotide delivery; oligonucleotides; precise polymers; sequence-defined polymers; siRNA delivery; solid-phase peptide synthesis; targeted delivery