Formulations for natural and peptide nucleic acids based on cationic polymeric submicron particles

Area of Science:

• Biomaterials Science
• Nanotechnology
• Gene Delivery Systems

Background:

• Developing effective nucleic acid delivery systems is crucial for gene therapy and molecular biology research.
• Existing delivery systems, such as liposomes, often face challenges with stability and efficiency.
• Submicron particles offer potential advantages due to their physicochemical stability and suitability for large-scale production.

Purpose of the Study:

• To produce and characterize novel cationic submicron particles for nucleic acid and peptide nucleic acid delivery.
• To evaluate the complexation ability and in vitro transfection efficiency of these particles.
• To compare the performance of these novel particles against commercially available transfection kits.

Main Methods:

• Cationic submicron particles were formulated using Eudragit RS 100 and cationic surfactants (DDAB18, DEBDA).
• Particles were characterized for size, size distribution, morphology, and zeta potential.
• In vitro gene delivery efficiency was assessed using a luciferase activity assay in cultured cells.

Main Results:

• The cationic submicron particles exhibited stable physicochemical properties suitable for long-term storage.
• Particles demonstrated effective complexation with DNA/DNA, DNA/peptide nucleic acid (PNA) hybrids, and PNA-DNA chimeras.
• In vitro studies revealed superior transfection efficiency compared to commercial liposome-based transfection kits.

Conclusions:

• Cationic submicron particles represent a promising, stable, and industrially scalable system for gene and nucleic acid delivery.
• These novel particles offer enhanced transfection efficiency, outperforming current liposomal formulations.
• The findings support the potential of these submicron particles for advanced therapeutic and research applications in gene delivery.