GALENUS - Genetic modification of the CFTR-Gene for Cystic Fibrosis

Cystic fibrosis is an autosomal recessive genetic disease caused by mutations of the cystic fibrosis transmembrane conductance regulator gene (CFTR). CFTR is an anion channel located at the apical membrane of epithelia and seems to be of importance for the ion- and watery content of secretions from exocrine glands. Novel genetic tools, like the use of zinc finger nucleases or transposons, are promising strategies for long-term correction of affected epithelia or stem cells.

The consortium aims to exploit these technologies in an animal model to develop inventive strategies to restore long-term function (i) of affected epithelia in the lung by transposon-based in vivo gene transfer to the lung, and (ii) of immune cells by ex vivo gene correction of hematopoietic stem cells.