Selective Treatment of Cancer, HIV, Other RNA Viruses and Genetically Related Diseases Using Therapeutic RNA Switches
Posted Nov 05 2012 7:00pm
Description of Invention:
Targeted therapy in cancer or viral infections is a challenge because the disease state manifests itself mainly through differences in the cell interior, for example in the form of the presence of a certain RNAs or proteins in the cytoplasm.
The technology consists of designed RNA switches that activate the RNA interference pathway only in the presence of a trigger RNA or DNA to which they bind, in order to knock down a chosen gene that is not necessarily related to the initial trigger.
This new approach can lead to a new type of drug that has the unique feature of selectively causing a biochemical effect (such as apoptosis) in cells that are infected by RNA viruses (such as HIV), as well as cancer cells. The RNA switch concept can be expanded to selectively treat other genetically related diseases.
Targeted therapeutic for viral infections, cancer stem cells, and genetically related diseases
Research tool to study cancer or viral infection
Fewer side effects because the therapeutic RNA-interference pathway is only activated by the RNA switch when it is intact and in its active conformation
Selectively kills cells infected by RNA viruses
Contains a minimal number of single stranded nucleotides, thus minimizing the effects of nucleases
Development Status: In vitro data available
Inventors: Bruce A Shapiro (NCI) Eckart HU Bindewald (NCI) Kirill A Afonin (NCI)
Grabow WW, et al. "RNA Nanotechnology in Nanomedicine," in Nanomedicine and Drug Delivery (Recent Advances in Nanoscience and Nanotechnology), ed. M Sebastian, et al. (New Jersey: Apple Academic Press, 2012), 208-220. [Book Chapter]
Severcan I, et al. "Computational and Experimental RNA Nanoparticle Design," in Automation in Genomics and Proteomics: An Engineering Case-Based Approach, ed. G Alterovitz , et al. (Hoboken: Wiley Publishing, 2009), 193-220. [Book Chapter]
Shapiro B, et al. "Protocols for the In silico Design of RNA Nanostructures," in Nanostructure Design Methods and Protocols, ed. E Gazit, R Nussinov. (Totowa, NJ: Humana Press, 2008), 93-115. [Book Chapter]
Collaborative Research Opportunity: The NCI Center for Cancer Research Nanobiology Program is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize therapeutic RNA switches. For collaboration opportunities, please contact John Hewes, Ph.D. at firstname.lastname@example.org .
For Licensing Information Please Contact: John Stansberry Ph.D. NIH Office of Technology Transfer 6011 Executive Blvd. Suite 325, Rockville, MD 20852 United States Email: email@example.com Phone: 301-435-5236 Fax: 301-402-0220