Silencing RNA (siRNA) is of considerable current interest in biological research because it can elicit potent, target specific knockdown of any mRNA, creating a useful and proven genetic surrogate tool. However, a key problem for effective application of this technology is specific delivery to targeted cells. The goal of this project is to develop a new generation of nanocarriers for efficient delivery of siRNA based on our recent discovery on nanoparticle-amphipol complexes. In the two year project period, we will systematically examine the chemical and structural properties of nanoparticles and their effects on siRNA delivery in cells. We will focus on fluorescent quantum dots as a discovery tool. If successful, the proposed work will aid in development of siRNA carriers for specific treatment of cancer.
Targeted and traceable delivery of siRNA with nanoparticle-amphipol Narrative siRNA has become one of most valuable tools for investigating gene function and holds great potential in treating complicated human diseases, such as cancer, neurological disorders, and cardiovascular disease. However, the key obstacle preventing its successful application in clinical studies is how to specifically deliver siRNA molecules to targeted cells, a problem that has plagued traditional antisense therapy for years. Our observation that quantum dot-amphipol complexes are remarkably efficient in siRNA delivery and our ability in nanoparticle functionalization help us design truly multifunctional nanostructures that integrate sensitive detection and imaging, high delivery efficiency, low toxicity, enhanced targeting specificity, tunable siRNA release kinetics, and the capability to protect siRNA from enzymatic degradation. This innovative technological platform could result in widespread application of siRNA in vitro and in vivo.
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