Cystic fibrosis: therapeutic strategies are multiplying.

Since the cloning of the defective gene in cystic fibrosis, much has been l earned on the function of CFTR and on the mechanisms regulating its express ion. Based on the current understanding of the processes involved in lung d isease progression, a number of approaches have been developed using gene t herapy and pharmacological agents. Several of these these agents have been reported to restitute a function to CFTR with specific mutations. Other mol ecules act on channels other than CFTR, and may be effective by bypassing C FTR itself. In the present review the various therapeutical strategies curr ently investigated are discussed. (C) 1998 Elsevier, Paris.