[Protein repair therapy in cystic fibrosis].
In the past few years since the cloning of the gene defective in cystic fibrosis, much has been learned on the function of the protein cystic fibrosis transmembrane conductance regulator (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 not only gene therapy, but also pharmacological agents. Several of these agents have been reported to restore function to CFTR with specific mutations.
Clément
Annick
A
Service de pneumologie pédiatrique Centre de la mucoviscidose Hôpital Armand-Trousseau 75571 Paris. annick.clement@trs.ap-hop-paris.fr
Tamalet
Aline
A
Fauroux
Brigitte
B
Epaud
Ralph
R
D003550:Cystic Fibrosis / Q000145:classification / Q000235:genetics* / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000187:drug effects* / Q000235:genetics*; D015870:Gene Expression / Q000187:drug effects; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D009154:Mutation
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator