AML is a complex heterogeneous disease characterized by different genetic aberration. NUP 98 encodes a nuclear pore complex protein (NPC), which participates in transportation of molecules between nucleus and cytoplasm. Translocations between this gene and other partners like NSD1 results in the expression of a rare oncofusion protein. FUS-ERG is another AML-driving oncofusion protein which results from t(16;21). This fusion protein targets hematopoietic regulators and has been associated with several types of leukemia. In my project focus is on using new approaches in silencing these fusion transcripts. Lipid nanoparticles have been established as potent means in delivering therapeutic molecules previously. By optimizing lipid nanoparticles composition and making them more durable, in-vivo and in-vitro, siRNA mediated gene silencing can be considered robust approach in silencing fusion transcripts.
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