Abstract
The lack of an appropriate intracellular delivery system for therapeutic nucleic acids (TNAs) is a major problem in molecular biology, biotechnology, and medicine. A relatively new class of highly symmetrical hyperbranched polymers, called dendrimers, shows promise for transporting small TNAs into both cells and target tissues. Dendrimers have intrinsic advantages for this purpose: their physico-chemical and biological properties can be controlled during synthesis, and they are able to transport large numbers of TNA molecules that can specifically suppress the expression of single or multiple targeted genes. Numerous chemical modifications of dendrimers extend the biocompatibility of synthetic materials and allow targeted vectors to be designed for particular therapeutic purposes. This review summarizes the latest experimental data and trends in the medical application of various types of dendrimers and dendrimer-based nanoconstructions as delivery systems for short small interfering RNAs (siRNAs) and microRNAs at the cell and organism levels. It provides an overview of the structural features of dendrimers, indicating their advantages over other types of TNA transporters.
Highlights
The targeted regulation of expression of therapeutically relevant genes using various types of nucleic acids (NAs) has been demonstrated in numerous in vitro and in vivo biological model systems [1,2,3]
The scope of the current review covers the design of synthetic delivery systems for small therapeutic RNAs based on dendrimer-related polymeric materials: dendrimers and/or dendrons, describing their efficiency in in vitro and in vivo models The description and comparison of dendrimer-based delivery vehicles with other non-viral vectors can be found in other reviews [21,22,23]
The scope of this review is to summarize experimental data on the use of dendrimers for delivering short therapeutic RNAs in vitro and in vivo
Summary
The targeted regulation of expression of therapeutically relevant genes using various types of nucleic acids (NAs) has been demonstrated in numerous in vitro and in vivo biological model systems [1,2,3]. RNA interference works by blocking expression of the targeted gene by arresting or destroying the messenger RNA via the native cell protein complex (RNA-inducedsilencing complex, RISC) bound to short regulatory RNAs that act as guides This mechanism is described in more detail in a series of reviews [6,7,8,9]. MicroRNAs and siRNAs, as RNA interference effectors, are therapeutically interesting because of their ability to overcome the major limitations of traditional low molecular weight drugs. The scope of the current review covers the design of synthetic delivery systems for small therapeutic RNAs based on dendrimer-related polymeric materials: dendrimers and/or dendrons (structural elements of dendrimers), describing their efficiency in in vitro and in vivo models The description and comparison of dendrimer-based delivery vehicles with other non-viral vectors can be found in other reviews [21,22,23]
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