Sequence-defined shuttles for targeted nucleic acid and protein delivery.
Molecular medicine opens into a space of novel specific therapeutic agents: intracellularly active drugs such as peptides, proteins or nucleic acids, which are not able to cross cell membranes and enter the intracellular space on their own. Through the development of cell-targeted shuttles for specific delivery, this restriction in delivery has the potential to be converted into an advantage. On the one hand, due to the multiple extra- and intracellular barriers, such carrier systems need to be multifunctional. On the other hand, they must be precise and reproducibly manufactured due to pharmaceutical reasons. Here we review the design of precise sequence-defined delivery carriers, including solid-phase synthesized peptides and nonpeptidic oligomers, or nucleotide-based carriers such as aptamers and origami nanoboxes.
Pharmaceutical Biotechnology, Center for System-Based Drug Research, & Center for Nanoscience (CeNS), Ludwig-Maximilians-University, Munich, Germany.
Ruth
R
Röder
Ernst
E
Wagner
D000818:Animals; D057846:Cell-Penetrating Peptides / Q000737:chemistry / Q000378:metabolism; D002626:Chemistry, Pharmaceutical; D004247:DNA / Q000737:chemistry / Q000235:genetics / Q000378:metabolism*; D004337:Drug Carriers; D016503:Drug Delivery Systems; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D006801:Humans; D011506:Proteins / Q000008:administration & dosage* / Q000737:chemistry / Q000378:metabolism; D012313:RNA / Q000737:chemistry / Q000235:genetics / Q000378:metabolism*
D057846:Cell-Penetrating Peptides; D004337:Drug Carriers; D011506:Proteins; D012313:RNA; D004247:DNA