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 can be targeted to acute myeloid leukaemia cells using an scFv targeting CD33. In RUNX1/ETO-expressing SKNO-1 cells the optimum untargeted PNP formulation was able to knockdown the RUNX1/ETO fusion protein by ˜60% at an mRNA level and ˜40% at a p…