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Kick-off Meeting
January 25th-27th, 2005
Munich/Kloster Ettal |
Start of a new cystic fibrosis (CF) gene therapy project funded by the European Commission
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Cystic fibrosis is an autosomal-recessive inherited disease, with an incidence of 1:3000 newborns in middle Europe. It is one of the most common monogenic diseases in our population. Mutations in the ?cystic fibrosis transmembrane conductance regulator? (CFTR) gene, localized on the long arm of chromosome 7, lead to a lacking function of the CFTR protein on the apical membrane of ciliated epithelial cells and serous cells of the submucosal glands in the airways. The water- and salt-content of the liquid protecting the airways is regulated by CFTR. CFTR is a chloride channel exchanging chloride ions between the cytoplasm and the airway lumen, but the crucial effect for water- and salt-content of airway secretion is based on an inhibition of the epithelial sodium channel ENaC. The missing function of CFTR causes an increased ENaC-mediated sodium uptake out of the luminal secretions of the airways, depleting the airways and leading to a defective mucociliar clearance. This benefits bacterial infections, resulting in a slow progredient destruction of the airway tissue. Approximately 90% of CF-patients die from pulmonal complications. The anticipated average life of CF-patients in middle Europe is 32 years.
Recently the European Commission approved a project investigating a new approach to the gene therapy of cystic fibrosis. It will be analyzed, to what extent the epithelial sodium channel ENaC can be downregulated by RNA interference using siRNA and if normal cell function can be recovered.
The technology of RNA interference has been successfully used in inhibiting the formation of gene products in vitro. During the last years the comprehension of different gene functions could be broadened and applied in the development of new therapies.
The European Commission supports the project over a period of 3 years in the context of the "Sixth Framework Programme for Research and Development" with 3.5 million Euro. Coordinator of the project is PD Dr. J. Rosenecker, Children's Hospital of the LMU at the Dr. von Haunersches Kinderspital. From January 25.-27 a kick-off meeting took place at the monastery of Ettal, which was attended by all participants. The working plans of the respective groups for the following months have been discussed. The project is characterized by the application of the promising technology of siRNA in an international surrounding. Totally 11 scientific groups from Germany, Italy, Great Britain, The Netherlands, France and Slovakia are involved, contributing their expertise in different parts of the project.
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