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Nucleic Acid Nanoparticles for Triggering RNA Interference

Summary
RNA interference (RNAi) is a naturally occurring cellular post-transcriptional gene regulation process that utilizes small double-stranded RNAs to trigger and guide gene silencing. By introducing synthetic RNA duplexes called small-interfering RNAs (siRNAs), we can harness the RNAi machinery for therapeutic gene control and the treatment of various diseases. The National Cancer Institute seeks partners to license or co-develop RNA, RNA-DNA, and DNA-RNA hybrid nanoparticles consisting of a DNA or RNA core with attached RNA or DNA hybrid duplexes.
NIH Reference Number
E-156-2014
Product Type
Keywords
  • RNAi
  • siRNA
  • nanotechnology
Collaboration Opportunity
This invention is available for licensing and co-development.
Contact
Description of Technology

RNA interference (RNAi) is a naturally occurring cellular post-transcriptional gene regulation process that utilizes small double-stranded RNAs to trigger and guide gene silencing. By introducing synthetic RNA duplexes called small-interfering RNAs (siRNAs), we can harness the RNAi machinery for therapeutic gene control and the treatment of various diseases.
 
NCI researchers created RNA, RNA-DNA, or DNA-RNA hybrid nanocubes consisting of a DNA or RNA core (composed of six strands) with attached RNA or DNA hybrid duplexes. The nanocubes can induce the reassociation of the RNA duplexes, which can then be processed by the human recombinant DICER enzyme, thus activating RNAi. This technology opens a new route for the development of “smart” nucleic acid based nanoparticles for a wide range of biomedical applications.  Immune responses can be controlled by altering the composition of the particle.

The researchers are conducting preliminary mouse xenograft studies on a related potential therapeutic, and seek collaborators for scale-up, animal models, developing particles for multiple targets, and RNAi delivery methods.

Potential Commercial Applications

• Treatment for cancer and infectious diseases.

Competitive Advantages

• Low cytotoxicity

Inventor(s)

Bruce A. Shapiro (NCI), Kirill A. Afonin (NCI), Mathias D. Viard (NCI)

Development Stage
Publications

Afonin KA, et al. [PMID 24189588]

Patent Status
  • U.S. Patent Filed: U.S. Patent Application Number PCT/US2015/029553
  • Foreign Filed: Foreign Filed - Patent Application WO 2010/1480
  • Foreign Filed: Foreign Filed - Patent Application WO 2013/075140
  • Foreign Filed: Foreign Filed - Patent Application WO 2014/039809
Therapeutic Area
Updated
Wednesday, August 31, 2016