[Mucoviscidosis: therapeutic strategies are multiplying].
Since the cloning of the defective gene in cystic fibrosis, much has been learned on the function of CFTR and on the mechanisms regulating its expression. Based on the current understanding of the processes involved in lung disease progression, a number of approaches have been developed using gene therapy and pharmacological agents. Several of these agents have been reported to restitute a function to CFTR with specific mutations. Other molecules act on channels other than CFTR, and may be effective by bypassing CFTR itself. In the present review the various therapeutical strategies currently investigated are discussed.
Clément
A
A
Département de pneumologie pédiatrique, hôpital Armand-Trousseau, Paris, France.
Tamalet
A
A
Fauroux
B
B
Tournier
G
G
D002648:Child; D003001:Cloning, Molecular; D003131:Combined Modality Therapy; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D015316:Genetic Therapy; D006801:Humans; D011379:Prognosis
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator
Mucoviscidose: les stratégies thérapeutiques se multiplient.