Aquaporin gene therapy corrects Sjogren's syndrome phenotype in mice. Homo sapiens

Primary Sjögren’s syndrome (pSS) is a chronic autoimmune disease that is estimated to affect 35 million people worldwide. Currently, no effective treatments exist for Sjögren’s syndrome, and there is a limited understanding of the physiological mechanisms associated with xerostomia and hyposalivation. The present work revealed that aquaporin 5 expression, a water channel critical for salivary gland fluid secretion, is regulated by bone morphogenetic protein 6. In- creased expression of this cytokine is strongly associated with the most common symptom of primary Sjögren’s syndrome, the loss of salivary gland function. This finding led us to develop a novel ther- apy in the treatment of Sjögren’s syndrome by increasing the water permeability of the gland to restore saliva flow. Our study demon- strates that the targeted increase of gland permeability not only resulted in the restoration of secretory gland function but also re- solved the hallmark salivary gland inflammation and systemic inflam- mation associated with disease. Secretory function also increased in the lacrimal gland, suggesting this local therapy could treat the sys- temic symptoms associated with primary Sjögren’s syndrome. Overall design: Human salivary gland cells were grown in culture in the presence or absence of BMP-6 at concentractions of 0.1 ng/mL, 6.0 ng/mL or 150 ng/mL. The study includes 12 samples, with 6 negative controls without BMP6 treatment and two biological replicates for each BMP6 treatment group. **Please note that the data (both raw and normalized data) have been updated on Jan 4, 2017 to fix the errors made during the initial submission**