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Content archived on 2024-05-27

Cystic fibrosis: rescue of the function and of the processing of cftr mutants by pharmacological agents and by interacting proteins (CF-PRONET)

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In search of new Cystic Fibrosis cures

The pathology of cystic fibrosis (CF) at the molecular level can be largely attributed to the dysfunction of a particular protein, the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

The CF-PRONET project concentrated on the design of novel compounds to correct the function defects of CFTR, which acts as a chloride ion channel on the cell surface. Furthermore, project partners worked on delineating the network of proteins interacting with CFTR and their potential in compensating for its dysfunction in CF patients. In the framework of the CF-PRONET project, University of Poitiers developed a reliable system to screen large numbers of compounds in terms of their ability to inhibit or activate CFTR. The university team focused on the development of four screening protocols based on the nature of CFTR. Wild-type CFTR as well as mutant versions were used. The end result was high throughput screening systems with the ability to isolate compounds active on each CFTR type. This research constitutes a crucial first step towards the discovery and subsequent development of new therapeutic agents for the treatment of CF. The university has already filed for patent protection and further tests are to be conducted before promising compounds are discovered and isolated. Pharmaceutical companies active in the field of CF are likely to be interested in furthering this line of research.

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