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CYSTIC FIBROSIS

EXPERIMENTAL CELL THERAPY AS TREATMENT FOR CHOLANGIOPATY ASSOCIATED TO CYSTIC FIBROSIS


Cystic Fibrosis is a common genetic disease with severe clinical implications, caused by mutations in the CFTR, a membrane protein that mediates the secretion of fluid and chlorine in many secretory epithelia, including bile ducts epithelium. The liver complications affect 30% of patients with cystic fibrosis and affect their survival and quality of life. A dysfunction of CFTR in biliary epithelium causes a chronic progressive colangytis which evolves in biliary cirrhosis.
Todate the terapy for this condition has proven to be proven unsatisfactory, limited to the administration of ursodeossicolic acid or transplantation in cases of progression. Cell transplantation and the organ repopulation by the healthy liver cells, is an attractive strategy to repair genetic defects of the liver. Preliminary studies have demonstrated the validity of this approach. Our assumptions and that, finding the ideal conditions for the selective growth and repopulation of transplanted cells, we will be able to drive the differentiation of transplanted hepatocytes and biliary cells thus repairing the defect in the secretory hepatic cystic fibrosis.
The aim of this project is to find the experimental conditions for the transplantation of liver cells as a strategy for the treatment of the cholangiopatia related to cystic fibrosis. These studies will enable us to increase the knowledge of the pathophysiology of the liver in cystic fibrosis and will be a proof of concept on the possibility of using cell therapy to treat cystic fibrosis related liver disease.

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