RNA/DNA Nanoparticles as Cancer Therapeutics

The development of RNA-based nanostructures and their use in a variety of applications, including RNA interference (RNAi) and drug delivery, represents an emerging field of science, technology, and biomedicine.  RNA is a dynamic material because of its natural functionalities, its ability to fold into complex small structures, and its capacity to self-assemble. 
Taking advantage of these characteristic, NCI Researchers have improved upon their existing invention of multi-functional RNA/DNA nanoparticles by adding a RNA toehold instead of a DNA toehold.  In in vitro studies, they have shown that the nanoparticles are capable of inhibiting HIV-1 gene expression in Hela cells transfected with a HIV-1 infectious clone and GFP expression in MDA-MB231 breast cancer cell lines transfected with eGFP. 

Competitive Advantages:

• Small size
• Chemical stability
• Less immunogenic

Commercial Applications:

• Therapeutic siRNA for cancer, CNS, and viral infections
• Diagnostic to visualize cancerous or virus-infected cells
• Increasing market for RNA-based therapeutics expected to continue by >25% annually through 2020