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Activation of Therapeutic Functionalities with Chimeric RNA/DNA Nanoparticles for Treatment of Cancer, Viruses and Other Disease

Posted Nov 05 2012 7:00pm

Description of Invention:
A new strategy based on RNA/DNA hybrid nanoparticles, which can be generally used for triggering multiple functionalities inside diseased cells is presented. Individually, each of the hybrids is functionally inactive and functional representation can only be activated by the re-association of at least two cognate hybrids simultaneously present in the same cell. Overall, this novel approach allows (i) the triggered release of therapeutic siRNAs or miRNAs inside the diseased cells, (ii) activation of other split functionalities (e.g. FRET, different aptamers, rybozymes, split proteins) intracellularly, (iii) higher control over targeting specificity (e.g. if two hybrids are decorated with two different tissue specific recognition moieties), (iv) biosensing and tracking of the delivery and re-association of these hybrids in real-time inside cells, (v) increasing the number of functionalities by introducing a branched hybrid structure, (vi) introduction of additional functionalities without direct interference of siRNA processivity, (vii) increasing the retention time in biological fluids by fine-tuning chemical stability through substituting the DNA strands with chemical analogs (e.g. LNA, PNA, etc.), (viii) conditional release of all functionalities.

  • Therapeutic siRNA for cancer, viruses and other diseases
  • Therapeutic for delivery of multiple functionalities
  • Diagnostic to visualize cancer cells, virus infected cells, or diseased cells, or track the delivery and effectiveness of siRNA treatment or other treatments associated with the particle
  • Research tool to study cancer, viral infections or other diseases

  • Novel way for multiple functionality delivery and activation
  • Enhanced chemical stability and pharmacokinetics due to the average size of nanoparticles exceeding 10nm
  • Increased specificity for selecting cells of interest using more than one target gene

Development Status:
  • In vitro data available
  • In vivo data available (animal)

Bruce A Shapiro (NCI)
Kirill A Afonin (NCI)

Patent Status:
HHS, Reference No. E-039-2012/0
US, Application No. 61/561,257 filed 17 Nov 2011

Related Technologies:
US, Application No. 61/561,247 filed 17 Nov 2011, Reference No. E-038-2012/0
US, Application No. 61/678,434 filed 01 Aug 2012, Reference No. E-038-2012/1

Relevant Publication:
  1. Afonin KA, et al. [ PMID 23016824 ]
  2. 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]
  3. Shukla GC, et al. [ PMID 21604810 ]
  4. Afonin KA, et al. [ PMID 22134126 ]
  5. Bindewald E, et al. [ PMID 22067111 ]
  6. Grabow WW, et al. [ PMID 21229999 ]
  7. Kasprzak W, et al. [ PMID 21163354 ]
  8. Afonin KA, et al. [ PMID 20802494 ]
  9. 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]
  10. 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]
  11. Bindewald E, et al. [ PMID 18838281 ]
  12. Yingling YG, Shapiro BA. [ PMID 17616164 ]

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/DNA nanoparticles. For collaboration opportunities, please contact John Hewes, Ph.D. at .

For Licensing Information Please Contact:
John Stansberry Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5236
Fax: 301-402-0220

Ref No: 2496

Updated: 11/2012

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