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Targeted delivery of pharmaceuticals to an intended site of action in the body is one of the most important issues for the next generation of therapeutics. Polymeric nanoparticles with surface-attached functionalities directed towards certain receptors/cell types can function as carriers for targeted drug or gene delivery. In gene therapy, DNA or RNA is delivered to the cell, inducing or suppressing a specific genetic function. An ideal gene delivery system should be capable to act as a synthetic virus, displaying high specificity for the target cells, protecting the polynucleotide from undesired interactions and degradation, and enhancing cell binding and intracellular delivery into cytoplasm and (for DNA) into nucleus (Figure 1). In our research tailor-made biodegradable and non-toxic polymers with desirable functional groups and properties are developed for innovative drug and gene delivery systems. The structural and physicochemical properties of the developed polymeric systems are closely correlated to their biological properties and targeting capabilities. These investigations form the basis for the development of efficient and prolonged drug and gene delivery systems.
Figure 1. Principle of gene therapy using polymeric gene carriers. Therapeutic polynucleotide (DNA or RNA) is mixed with a multifunctional, biodegradable, cationic polymer to form polyplex particles that can enter target cells by a process called endocytosis. The properties of the polymer must be such that release of polynucleotide from the endosomes and (for DNA) entrance to the cell nucleus is promoted, while protecting the polynucleotide from degradation by lysosomes.