POLYPLEX FORMATION BETWEEN CYCLODEXTRIN-BASED NON-VIRAL VECTOR AND dsDNA: MOLECULAR DYNAMIC STUDY WITH EXPERIMENTAL VALIDATION

Theoretical and experimental studies of the polyplex formation between nucleic acids and synthetic non-viral vectors have received tremendous attention in the last years owing to the emerging application of synthetic vectors in gene therapy. Molecular dynamics simulation is a computational tool useful for understanding the structures and functions of biological macromolecules as well as their interactions. In our study we report in silico simulations and experimental evidences regarding the polyplex formation between short double-stranded DNA and beta-cyclodextrin-based non-viral vector functionalized with poly(ethylene imine). The data have shown that the synthetic vector is able to efficiently bind DNA, one molecule being able to bind multiple DNA molecules at physiological pH. Theoretical studies have been supported by the experimental evidences of polyplex formation by Gel Red staining assay, proving the ability of the non-viral vector to efficiently bind DNA and hint to a feasible application in gene therapy.