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Novel Protein Delivery System for Mammalian Cells

Posted Feb 01 2009 4:00pm

Description of Invention:
Virus-like particles (VLPs) consist of viral structural proteins that are capable of self-assembly into a nanoparticle, but are non-infectious because they lack viral nucleic acids. VLPs have been used in viral vaccines, such as those for human papilloma virus and hepatitis B. However, they also have great potential in other applications, such as cancer vaccines, transport of nucleic acids into target cells (gene therapy), and transport of biologics or other large molecules into target cells for therapeutic purposes. The present technology discloses a chimeric VLP containing a GAG-Cre recombinase fusion protein. This recombinase fusion protein retains Cre recombinase activity, and can excise a LOX-flanked gene in a transduced target cell. Experiments by Drs. Kaczmarczyk and Chatterjee have demonstrated that chimeric VLPs can be used to deliver functional fusion proteins into cells. The technology also provides for a two-VLP protein delivery system designed to deliver a protein of interest into a target cell. The present technology also discloses VLPs containing GAG-protein of interest (ex. GAG-Cre) co-packaged with GAG-protease to deliver protein of interest in target site as a fully-processed protein rather than as a fusion protein.

The claims in the pending patent application provide for virus like particles, methods of making virus like particles, and methods of using virus like particles to deliver proteins to a cell. The claims also provide for methods of targeting a protein to a cell, methods of protein therapy and methods of treating diseases or disorders.

  • Intracellular targeted delivery of therapeutic proteins.
  • Ex vivo use for expansion of stem cells for transplantation.
  • Antigen loading of dendritic cells for cancer vaccination.

Development Status:
In-vivo feasibility studies are in progress.

Stanislaw J Kaczmarczyk (NCI)
Deb K Chatterjee (NCI)

Patent Status:
HHS, Reference No. E-010-2008/0
PCT, Application No. PCT/US2009/59328 filed 02 Oct 2009

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The National Cancer Institute Advanced Technology Program, Protein Expression Laboratory, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize this technology. Please contact John D. Hewes, Ph.D. at 301-435-3121 or for more information.

Devices/Instrumentation - Research Tools and Materials
Gene Based Therapies
Gene Based Therapies - Therapeutics
Research Materials

For Additional Information Please Contact:
Suryanarayana Vepa Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5020
Fax: 301-402-0220

Ref No: 1882

Updated: 02/2009

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