[Cystic fibrosis: chemical drugs to cure genetic diseases]. / Mucoviscidose: y a-t-il des alternatives aux thérapies géniques?

ABSTRACT

Cystic Fibrosis is the most common lethal genetic disease among Caucasian population. Despite considerable efforts, no significant progress has been so far achieved by gene therapies approaches. On the basis of a surprising clinical observation, we have developed an approach using anti-cancer drugs promoting the over expression of ABC transporters closely related to the deficient protein CFTR, which seem able to share functions with it and to restore the missing function(s).