title: To be targeted: is the magic bullet concept a viable option for synthetic nucleic acid therapeutics?
abstract: Nucleic acids offer the possibility of tailor-made, individualized treatments for genetic disorders, infectious diseases, and cancer. As an alternative to viral vectors, synthetic delivery systems have a potentially improved safety profile, but often lack sufficient efficiency especially when applied in vivo. Receptor targeting of synthetic vectors can improve the specificity of the vector and increase the efficiency of nucleic acid delivery to the target site. This review covers recent concepts for targeted DNA and RNA delivery to organs like liver and lung, and also to solid cancers. Syntheses and applications of delivery systems targeted with proteins, peptides, and small molecules as ligands coupled to polymeric or lipidic nucleic acid carriers are reviewed. Therapeutic concepts for treatment of genetic and infectious diseases are explained. Systemic treatment regimens of metastasized malignancies in combination with chemotherapy and radiation have already been successfully applied in preclinical studies. In addition, a first clinical study in the human application of a targeted synthetic carrier has been performed.
authors.affiliation: Pharmaceutical Biotechnology, Ludwig Maximilians University, Munich 81377, Germany. manfred.ogris@cup.uni-muenchen.de
authors.forename: Manfred
authors.initials: M
authors.lastname: Ogris
authors.forename: Ernst
authors.initials: E
authors.lastname: Wagner
mesh_terms: D004337:Drug Carriers / Q000008:administration & dosage; D016503:Drug Delivery Systems / Q000379:methods*; D015316:Genetic Therapy; D006801:Humans; D008024:Ligands; D008055:Lipids / Q000008:administration & dosage / Q000235:genetics; D009369:Neoplasms / Q000188:drug therapy / Q000235:genetics; D009696:Nucleic Acids / Q000008:administration & dosage* / Q000235:genetics*; D010455:Peptides / Q000008:administration & dosage / Q000235:genetics; D011108:Polymers / Q000008:administration & dosage; D034741:RNA, Small Interfering / Q000008:administration & dosage
chemical_list: D004337:Drug Carriers; D008024:Ligands; D008055:Lipids; D009696:Nucleic Acids; D010455:Peptides; D011108:Polymers; D034741:RNA, Small Interfering