The transfer and expression (transfection) of genetic material into specific cells of a patient in order either to replace a defective gene or to introduce a new function to the cells can be performed with multiple delivery technologies. Synthetic transfection systems offer a promising alternative to viral vectors due to their good safety profile, ease of manufacturing and storage under GMP-compliant norms and limitless size of the nucleic acid material that can be delivered. However, the efficiency of these systems has to be improved, by adapting the structure of the lipoplexes to the extracellular and intracellular delivery barriers.We will present our results towards the formulation, DNA compaction properties and stability of a DNA delivery system based on a novel biodegradable pyridinium cationic lipid, designed to provide more flexible cationic bilayers with enhanced fusogenicity.

Novel pyridinium lipoplexes for nucleic acid delivery – a formulation and stability study

244th National Meeting (2012)

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