Gene Therapy for Reflux Disease in the Esophagus


Almost one-third of adults in the US experience symptoms of gastroesophageal reflux disease (GERD) and patients with chronic GERD have an increased risk for the development of esophageal adenocarcinoma. Although proton pump inhibitors (PPIs) have shown effective treatment for GERD, up to 45% of patients with GERD remain symptomatic on standard therapy and long-term use of PPIs can cause severe side effects in the lung and heart. There is an urgent need to develop new methods to treat refractory GERD. The esophageal epithelium tissues of patients with GERD have a special pathological feature of barrier disruption which causes chronic inflammation and tissue damage. The restoration of this barrier function is a future promising therapeutic target to prevent the disease progression. 

Technology Overview

Researchers at the Univesity of Rochester have developed a novel electroporation-mediated gene delivery method to transfer Na, K-ATPase beta1 subunit in the esophagus. The researchers have reported previously that gene transfer of the ß1 subunit of the Na+, K+-ATPase results in the upregulation of tight and adherens junction proteins and increases barrier function in cultured cells and living animals. The method represents future therapy to treat refractory GERD and further prevent disease progression to esophageal cancer.


The gene delivery method has been shown safe and efficient when tested in the lungs of living animals. Gene transfer of Na+, K+-ATPase beta1 subunit in the esophagus resulted in the prevention breakdown of barrier functional complexes between the cells and complete prevention of pathological tissues of GERD in rabbit models.


Therapeutic for refractory GERD

URV Reference Number: 6-23001
Patent Information:
For Information, Contact:
Liliam Martinez Bello
Licensing Manager
University of Rochester
David Dean
Zhongren (David) Zhou