- Therapeutics
- Jasmine Yang
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.
- 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
Bruce Shapiro (NCI)
- Pre-clinical (in vivo)
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]
- 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
- E-059-2009 - In silico design of RNA nanoparticles
- E-038-2012
- E-039-2012 - Nanoparticles for the targeted treatment of infected cells
- E-765-2013 - Multifunctional RNA Nanoparticles as Cancer and HIV Therapeutics
- E-156-2014 - Nucleic Acid Nanoparticles for Triggering RNA Interference
- E-277-2016 - Functionally-Interdependent Shape-Switching Nucleic Acid Nanoparticles
- Cancer/Neoplasm