Technology ID

Nucleic Acid Nanoparticles for Triggering RNA Interference

Linked ID
Bruce Shapiro (NCI)
Kirill Afonin ()
Mathias Viard ()
Lead Inventors
Bruce Shapiro (NCI)
Kirill Afonin ()
Mathias Viard ()
Development Stages
Pre-clinical (in vivo)
Development Status
Pre-clinical (in vivo)
Commercial Applications

• Treatment for cancer and infectious diseases.

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 areconducting preliminary mouse xenograft studies on a related potential therapeutic, andseek collaborators for scale-up, animal models, developing particles for multiple targets, and RNAi deliverymethods.
Competitive Advantages

• Low cytotoxicity

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