Synthetic diblock copolymer micelles as vectors for siRNA delivery to knockdown RUNX1/ETO in acute myeloid leukaemia cells

Knockdown of RUNX1/ETO using short interfering ribonucleic acids (siRNAs) in vitro has been shown to inhibit acute myeloid leukaemia (AML) cell growth and proliferation. Due to the poor pharmacokinetic properties of siRNA a delivery system is required for therapeutic use in vivo. We propose a polymeric nanoparticle (PNP) system consisting of two diblock copolymers which allow for rapid determination of structure-activity relationships involving gene knockdown and toxicity using a luciferase reporter assay. This high throughput screening method allows rapid determination of the optimum PNP formulation under speci…