Targeted RNA/DNA Nanoparticles with Single Stranded RNA Toeholds

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. 

Potential Commercial Applications: Competitive Advantages:
  • 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
  • Small size
  • Chemical stability
  • Less immunogenic

Development Stage:
Pre-clinical (in vivo)

Related Invention(s):


Bruce Shapiro (NCI)  ➽ more inventions...

Intellectual Property:
Application No. PCT/US2016/017661

Afonin K, et al. The use of minimal RNA toeholds to trigger the activation of multiple functionalities.   PMID: 26926382
Bindewald E, et al. Multistrand structure prediction of nucleic acid assemblies and design of RNA switches.  PMID: 26926528

Collaboration Opportunity:

Licensing and research collaboration

Licensing Contact:
John Hewes, Ph.D.
Phone: 240-276-5515

OTT Reference No: E-078-2016
Updated: Jul 27, 2018