Technology Bundle ID
TAB-2685

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

Linked ID
E-139-2011-1
Lead Inventors
John Chiorini (NHLBI)
Development Stages
Development Status
  • Pre-clinical
  • In vitro data available
  • In vivo data available (animal)
ICs
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.
Commercial Applications
  • Prevention of dry mouth (xerostomia) associated with salivary gland dysfunction in patients with Sjögren's syndrome.
Competitive Advantages
  • AAV gene transfer to salivary glands is highly efficient.
  • AAV-AQP1 promotes de novo salivary flow.

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