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RNA/DNA Nanoparticles as Cancer Therapeutics

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The technology is directed to the use of single-stranded RNA overhangs or toeholds of varying lengths (< 12 nucleotides) contained in nucleic acid-based nanoparticles which trigger the association of these nanoparticles and activates multiple functionalities such as gene silencing and/or cell-specific targeting. The use of RNA toeholds is superior to that of DNA toeholds in that it allows for smaller nanoparticles (fewer nucleotides for the toeholds) resulting in greater chemical stability, less immunogenic and higher yield of production. The National Cancer Institute (NCI) seeks licensing and/or co-development research collaborations for use of RNA overhangs or toeholds in nucleic acid nanoparticles.
NIH Reference Number
Product Type
  • Gene Silencing, Nanobiology, RNAi, Shapiro
Collaboration Opportunity
This invention is available for licensing and co-development.
Description of Technology

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
  • 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
Competitive Advantages
  • Small size
  • Chemical stability
  • Less immunogenic

Bruce Shapiro (NCI)

Development Stage

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]

Patent Status
  • PCT: PCT Application Number PCT/US2017/017661 , Filed 13 Feb 2017
  • U.S. Patent Filed: U.S. Patent Application Number US16/076,878, Filed 09 Aug 2018
  • Foreign Filed: - Patent Application 17706653.7, Filed 12 Sep 2018
  • U.S. Provisional: U.S. Provisional Patent Application Number 17/102,786, Filed 27 Nov 2020
Therapeutic Area
Monday, December 5, 2022