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AAV-Aquaporin-1 Gene Therapy for Sjogren's Syndrome

19:00 EST 2 Dec 2013 | NIH

Sjögren's syndrome is a chronic inflammatory disease affecting over 2 million Americans, whereby moisture-producing glands are attacked by the body's immune system. The disease is marked by disabling dryness of the mouth and eyes as well as fatigue and pain. Researchers at the National Institute of Dental and Craniofacial Research have developed a therapy that alleviates xerostomia in an animal model of Sjögren's syndrome. This technology consists of local delivery of adeno-associated virus (AAV) mediated aquaporin-1 (AQP1) fusion protein to salivary glands. Using a murine model that mimics Sjögren's dry mouth symptoms, it was discovered that treatment restored salivary fluid movement upon expression of AQP1. Targeted delivery of the AAV-AQP1 system makes this invention a novel and potential long-term therapeutic for restoration of exocrine gland function and prevention of xerostomia-associated pain associated with Sjögren's syndrome.

IC: 
NHLBI
NIH Ref. No.: 
E-139-2011-1
Advantages: 
  • AAV gene transfer to salivary glands is highly efficient.
  • AAV-AQP1 promotes de novo salivary flow.
Applications: 
  • Prevention of dry mouth (xerostomia) associated with salivary gland dysfunction in patients with Sjögren's syndrome.
Development Status: 
  • Pre-clinical
  • In vitro data available
  • In vivo data available (animal)
Provider Technology ID: 
2685
Updated On: 
May 1, 2019
Date Published: 
Tuesday, December 3, 2013
Patent Application: 
61/695,753
PCT/US13/57632
14/423,774
Patent Authority: 
US
PCT
US
Lead Inventor: 
Inventor IC: 
NHLBI
Inventor Lab URL: 
https://irp.nih.gov/pi/john-chiorini
LPM FIrst Name: 
Vladimir
LPM Last Name: 
Knezevic
LPM Address: 
BG 12A RM 3011 12 SOUTH DR
LPM City: 
Bethesda
LPM Zip: 
20892
Inv Is lead: 
LPM State: 
MD
LPM Phone: 
301.443.5560
LPM Suffix: 
M.D.
LPM Organization: 
NIH Office of Technology Transfer
DTDT Classification: 
Gene Based Therapies
In vitro data available
In vivo data available (animal)
DTDT Description: 
Gene Based Therapies
In vitro data available
In vivo data available (animal)
Pat Filing Date: 
2012-08-31
2013-08-30
2015-02-25
Collaboration Sought: 
Yes
Institute or Center: 
Collaboration Opportunity: 

The National Institute of Dental and Craniofacial Research, AAV Biology Section, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize AAV-Aquaporin-1 Gene Therapy for Sjögren's. For collaboration opportunities, please contact David Bradley at bradleyda@nidcr.nih.gov.

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Related Patent Authority: 
US
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Related Invention: 
E-127-1998-0
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E Number Only: 
E-139-2011
Inventor First Name: 
John
Inventor Last Name: 
Chiorini

Original Article: AAV-Aquaporin-1 Gene Therapy for Sjogren's Syndrome

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