Polymeric nanoparticles for siRNA delivery: Production and applications

Rekindling RNAi Therapy: Materials Design Requirements for In Vivo siRNA Delivery

“…Based on published data accumulated over the past decade, average gene silencing efficiencies of 64.6 ± 24.7% in vitro and 61.5 ± 19.6% in vivo have been achieved (Figure and Table ). The three most frequently employed types of polymer nanoparticles for siRNA delivery have been solid polymeric nanoparticles, dendrimers, and hydrogels, but across the wide range of structural designs in polymeric delivery systems, there are a number of proven components that are regularly used: poly(lactic‐co‐glycolic acid) (PLGA), poly‐L‐lysine (PLL), chitosan, and polyethyleneimine (PEI) …”

“…PLL's primary advantage is in its relatively high biocompatibility compared to the fairly toxic PEI, but PLL systems suffer from diminished transfection efficiency in the presence of high serum content; in an environment that is clinically relevant and reflective of our vasculature (which has high serum content), the PLL systems have difficulty forming stable structures due to competition with serum proteins (which are generally anionic) for binding to the siRNA payloads . Thus, recent efforts have focused on developing PPL derivatives and co‐polymers to reinforce their vulnerability to high serum content environments . Lastly, chitosan is a cationic polysaccharide, which offers high biocompatibility and many amine and hydroxyl groups available for chemical modification.…”

Applications of Lipidic and Polymeric Nanoparticles for siRNA Delivery

“…Polysaccharides are generally biocompatible polymers. The main advantage is the presence of different functional groups (i.e., carboxyl, hydroxyl, amine) which enable functionalization to obtain structural heterogeneity and copolymers [54].…”

Polymer-Based Systems for Controlled Release and Targeting of Drugs

“…

The current need to find new advanced approaches to carry biologically active substances (conventional organic drugs, peptides, proteins (such as antibodies), and nucleic acid-based drugs (NABDs such as siRNA and miRNA)) in the body fluids, to realize targeted therapies and even personalized ones, goes hand in hand with research on the performance of new materials to better realize appropriate drug vectors [1].Polymeric materials can be designed and manufactured to obtain delivery systems with the appropriate characteristics in terms of drug release and performance [2]. For use in human applications, the polymer must primarily be biocompatible and non-toxic, and then functionalizable to give the appropriate structural and functional characteristics, such as to make it easily workable, processed, and engineered to obtain the desired system, and to be applied in drug delivery and targeting and/or in diagnosis of diseases.The further possibility of decorating the surface of these polymeric systems (due to the characteristics of the material that constitutes the matrix) with ligands capable of interacting specifically with membrane receptors on cells represents a unique advantage for obtaining targeted drug release to a specific organ, tissue, or cell type [3][4][5][6][7].In this issue, some current examples of design and production of polymeric materials, as well as of searching strategies to modify existing ones, for the making of innovative systems for drug delivery and/or regenerative medicine are collected.In particular, polymeric systems from nanoscale (micelles [8,9], nanoparticles [10,11]) to microscale structures (microparticles [12,13]), and to macrodevices (hydrogels [14] and films [15]) were produced.

…”

“…The current need to find new advanced approaches to carry biologically active substances (conventional organic drugs, peptides, proteins (such as antibodies), and nucleic acid-based drugs (NABDs such as siRNA and miRNA)) in the body fluids, to realize targeted therapies and even personalized ones, goes hand in hand with research on the performance of new materials to better realize appropriate drug vectors [1].…”