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A Novel and Efficient Technology for Targeted Delivery of siRNA

Posted Jun 15 2010 5:00pm

Description of Invention:
The biological phenomenon of RNA interference (RNAi) has much promise for developing therapeutics to a variety of diseases. However, development of RNAi therapies remains mainly in preclinical stages largely because of difficulties in delivering small inhibitory RNAs (siRNA) and short hairpin RNAs (shRNA) into target cells. Although viral vector-based siRNA delivery systems have been widely used, their specificity and safety remains significant issue. Without a solution to this delivery problem, RNAi cannot fulfill its therapeutic promise.

Investigators at the National Institutes of Health have developed novel compositions and methods for delivering inhibitory oligonucleotides to cells in a targeted and efficient manner. The compositions and methods are based on utilizing a cell surface receptor targeting ligand, such as cytokine or chemokine, and a domain that binds an inhibitory oligonucleotide, to efficiently deliver the inhibitory oligonucleotide to the cell that expresses the cell surface receptor targeting ligand. Chemokine receptors are differentially expressed on various cells, including tumors; hence this technology allows targeting siRNA to aberrant cells. Gene silencing can also be achieved in variety of immune cells by targeting cytokine receptors. This technology has great potential for developing into a safe and effective means of delivering therapeutic siRNAs.

  • Treatment of cancers and autoimmune diseases by delivery of siRNA to tumor cells or various aberrantly functioning immune cells.
  • This technology can be used to boost vaccine responses against cancers and chronic infectious diseases.
  • Targeted delivery of fluorochrome-labeled RNA both in vitro and in vivo for diagnostic purposes, for example, to trace or localize various cells and to determine tumor metastasis and aberrant proliferation or homing of immune cells.

  • Simple method for linking siRNA to polypeptides to create non-covalent or covalent complexes
  • In vivo targeted delivery of inhibitory RNAs into cells rather than systemically
  • Delivery of multiple inhibitory RNAs to target multiple genes
  • Long term repression of target gene expression through RNAi phenomenon

Development Status:
Currently animal model studies planned

Arya Biragyn (NIA)
Purevdorj B Olkhanud (NIA)
Juan C Espinoza (NIA)

Patent Status:
HHS, Reference No. E-051-2008/0
PCT, Application No. PCT/US2009/040607 filed 15 Apr 2009

Relevant Publication:
  1. None directly related to the invention.

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The National Institute on Aging, Immunotherapeutics Unit, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize chemokine-based siRNA/shRNA technology for treatment of cancers and autoimmune diseases, i.e. to control expression of immunomodulatory cytokines and other factors that facilitate tumor escape, activity of regulatory T cells or Th2 type of cells. This technology can be also utilized to boost vaccine responses against cancers and chronic infectious diseases. Please contact John D. Hewes, Ph.D. at 301-435-3121 or for more information.

Cancer - Therapeutics
Gene Based Therapies
Gene Based Therapies - Diagnostics
Gene Based Therapies - Therapeutics
In-vitro Data

For Additional Information Please Contact:
Patrick McCue Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-496-7057
Fax: 301-402-0220

Ref No: 1868

Updated: 06/2010

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