Nucleic Acid Nanoparticles for Triggering RNA Interference


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: Competitive Advantages:

• Treatment for cancer and infectious diseases.

 

• Low cytotoxicity



Development Stage:
Pre-clinical (in vivo)

Related Invention(s):
E-765-2013
E-039-2012
E-078-2016


Inventors:

Bruce Shapiro (NCI)  ➽ more inventions...

Kirill Afonin (NCI)  ➽ more inventions...

Mathias Viard (NCI)  ➽ more inventions...


Intellectual Property:
U.S. Filed Application No. PCT/US2015/029553
Foreign Filed Application No. WO 2010/1480
Foreign Filed Application No. WO 2013/075140
Foreign Filed Application No. WO 2014/039809

Publications:
Afonin KA, et al. PMID 24189588

Collaboration Opportunity:

Licensing and research collaboration


Licensing Contact:
John Hewes, Ph.D.
Email: John.Hewes@nih.gov
Phone: 240-276-5515

OTT Reference No: E-156-2014
Updated: Aug 31, 2016