Probing the interaction of miRNA-loaded fluorinated dendrimer with model membranes: cellular and endosomal
Gene alteration is responsible for several complex illnesses. Gene therapy, working on altering, modifying, or silencing defective or mutated genes, has emerged as a promising therapeutic strategy. Dendritic-based vectors are a good candidate for gene delivery due to their well-defined chemical structure, easy tunability of density of terminal groups, and easy surface modification. The addition of fluorinated chains enhances serum stability of dendriplexes (complexes between dendritic vectors and genes), facilitates endosomal escape, enhances cellular uptake and reduces cytotoxicity. We combined Bis-MPA-based dendrimers with fluorination to improve their efficacy as gene vectors. We selected branched fluorinated moiety bearing three perfluoro-t-butoxyl groups as hydrophobic part and synthetized a fluorinated Janus-type dendrimer (FJD2N). The presence of four primary ammonium groups allows complexation with nucleic acids and has the potential to enhance endosomal escape. We aim to understand the mechanism of interaction of miRNA-FJD2N with cellular and endosomal model membranes for the development of more efficient gene delivery systems.
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SEBASTIANI Federica; Baldelli Bombelli Francesca; Nicoḷ Paracini and WACKLIN KNECHT Hanna. (2023). Probing the interaction of miRNA-loaded fluorinated dendrimer with model membranes: cellular and endosomal. Institut Laue-Langevin (ILL) doi:10.5291/ILL-DATA.9-13-1085
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