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AAV Mediated CTLA-4 Gene Transfer to Treat Sjogren's Syndrome

Posted Nov 17 2011 7:00pm

Description of Invention:
Sjögren’s syndrome is an autoimmune disease that affects over 2 million Americans, primarily over the age of 40. One of the major outcomes of Sjögren's syndrome is xerostomia (dry mouth) that is caused by immune system attack on moisture producing salivary glands. Researchers at the National Institute of Dental and Craniofacial Research have developed a therapy that alleviates xerostomia in a murine model of Sjögren's syndrome. This technology consists of a local delivery of adeno-associated virus (AAV) mediated cytotoxic T-lymphocyte antigen 4 Immunoglobulin-G (CTLA4IgG) fusion protein to salivary glands. The system effectively blocks CTLA4 ligand interactions with T cell surface receptors, resulting in immune suppression and reversal of autoimmune-related xerostomia. Targeted delivery of the AAV-CTLA4-IgG system makes this invention a novel therapeutic for the prevention of xerostomia-associated pain and discomfort caused by Sjögren's syndrome.

Prevention of salivary gland destruction and xerostomia development in patients with Sjögren's syndrome.

  • Current treatments temporarily reduce the discomfort of xerostomia but do not prevent the deleterious effects of this disorder.
  • AAV gene transfer to salivary glands is highly efficient.
  • AAV therapy is safe and noninflammatory.

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

Hongen Yin (NIDCR)
John A Chiorini (NIDCR)

Patent Status:
HHS, Reference No. E-087-2011/0
US, Application No. 61/476,168 filed 15 Apr 2011

Relevant Publication:
  1. Zheng C, et al. [ PMID 21625005 ]
  2. Kanaya K, et al. [ PMID 12589145 ]
  3. Genovese MC, et al. [ PMID 16162882 ]

Collaborative Research Opportunity:
The NIDCR is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize this technology. For collaboration opportunities, please contact David Bradley at .

For Licensing Information Please Contact:
Jaime Greene M.S.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5559
Fax: 301-402-0220

Ref No: 2339

Updated: 11/2011

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