Functional Gene Delivery via Structural Nucleic Acid Nanotechnology

PI’s: Yonggang Ke & Cheol Hee Cho

We want to develop novel, general platforms based on structural nucleic acid nanotechnology for functional in vitro/vivo gene delivery. In our new paradigm, the delivery targets themselves will fold into rational-designed nanoscale structures made purely of nucleic acids, without usage of nanoparticles or adenovirus. For this pilot project, we selected enhanced green fluorescence protein (eGFP) gene as the delivery target to study the idea’s feasibility and to understand important design/experimental strategies. Using the eGFP model system, we have successfully realized gene delivery to H1299 cancer cells using DNA nanostructure and proven the gene is active (figure). We expect our method will open a new avenue for gene therapy to cure children.