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Insect Cell Production of Recombinant Adeno-Associated Virus that Produce Cytotoxic Gene Products and Applications for Solid Tum

Posted Jun 15 2010 5:00pm

Description of Invention:
Cancer is the second leading cause of death in United States and it is estimated that there will be approximately 600,000 deaths caused by cancer in 2006. Due to the high incidence of death from cancer despite the use of current therapies, there is a strong need for targeted therapeutic approaches such as gene therapy.

This technology describes a new method for targeting solid tumors using gene therapy. More specifically, mammalian HEC-1 has a critical role in chromosome segregation and thus cell division. This technology involves targeted depletion of HEC-1 using shRNA against the HEC-1 mRNA inhibiting cancer cell growth in cell culture models (in vitro) as well as regressed tumor size in mouse model (in vivo). Additionally, this is the sole technology using an insect cell based recombinant adeno-associated virus (rAAV) gene transfer vehicle with high titer containing the shRNA of interest thus enabling high dosing during therapeutic intervention if necessary. This technology platform has the potential to treat a broad spectrum of cancers and related diseases.

  • A new anti-cancer adjuvant therapy for non-resectable tumors targeting HEC-1 protein
  • A new method involving insect cell based production of recombinant adeno-associated virus (rAAV) gene transfer vehicle

Development Status:
The technology is currently in pre-clinical stage of development.

Robert M Kotin (NHLBI)
Lina Li (NHLBI)

Patent Status:
HHS, Reference No. E-200-2005/0

Relevant Publication:
  1. EN Gurzov et al., "RNA Interference against Hec 1 inhibits tumor growth in vivo," Gene Ther. 2006 Jan; 13 (1):1-7.
  2. JG DeLuca et al., "Hec1 and nuf2 are core components of the kinetochore outer plate essential for organizing microtubule attachment sites," Mol Biol Cell. 2005 Feb; 16 (2):519-531.
  3. S Martin-Lluesma et al., "Role of Hec1 in spindle checkpoint signaling and kinetochore recruitment of Mad1/Mad2," Science 2002 Sep 27; 297 (5590):2267-2270.
  4. T Hori et al., "Dynamic behavior of Nuf2-Hec1 complex that localizes to the centrosome and centromere and is essential for mitotic progression in vertebrate cells," J Cell Sci. 2003 Aug 15; 116 (Pt 16):3347-3362.
  5. Y Chen et al., "Phosphorylation of the mitotic regulator protein Hec1 by Nek2 kinase is essential for faithful chromosome segregation," J Biol Chem. 2002 Dec 20; 277 (51):49408-49416.

Licensing Status:
Available for non-exclusive or exclusive licensing.

Collaborative Research Opportunity:
The National Heart, Lung, and Blood Institute, Laboratory of Biochemical Genetics, is seeking statements of capability or interest from parties interested in collaborative research to further develop therapeutics using rAAV-shRNA to induce selective cytotoxicity in primary and metastatic solid tumors. Partners are sought for conducting translational research from preclinical trials to clinical trials. Please contact Dr. Vincent Kolesnitchenko, Office of Technology Transfer and Development, NHLBI at 301-594-4115 or by e-mail ( for more information.

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

For Additional Information Please Contact:
Betty Tong Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-594-6565
Fax: 301-402-0220

Ref No: 1333

Updated: 06/2010

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