Berlin 2018 – wissenschaftliches Programm

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BP: Fachverband Biologische Physik

BP 15: Postersession III

BP 15.10: Poster

Dienstag, 13. März 2018, 14:00–16:00, Poster B

Lipid nucleic acid nanoparticles (LNPs) for delivery of single-stranded antisense oligonucleotides and targeted gene silencing — •Nicola Kerschbaumer, Rafal Krzyszton, and Joachim Rädler — Department of Physics, Ludwig-Maximilians-Universität (LMU) Munich, Geschwister-Scholl-Platz 1, 80539 Munich, Germany

Antisense oligonucleotides for gene silencing present a promising therapeutic strategy. Transfer of antisense oligonucleotides across cell membranes is limited and the development of an efficient and safe encapsulation of such antisense oligonucleotides for specific delivery becomes increasingly desirable. In previous work mononucleic acid lipid particles (mNALPs) were shown to form nanoparticles which self-assemble in a microfluidic setup when placing the lipids DOTAP, DOPE, DOPC, and DSPE-PEG2000 in a solvent solution with the usage of water as a buffer. Here we show that the same assembly strategy using microfluidic chips forms antisense LNPs with high encapsulation efficiency. The particles have 30 - 40 nm in diameter for 15 and 21 base-antisense oligonucleotides and are stable in blood serum over a period of several days as characterized using fluorescence correlation spectroscopy. We demonstrate that the particles bind specifically to cells expressing folate receptors and analyze their capability to silence targeted gene expression. Our LNP carrier provides a reasonable and effective approach for targeted delivery of single-stranded oligonucleotides for gene silencing.