[Cystic fibrosis: chemical drugs to cure genetic diseases].
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).
Lallemand
Jean-Yves
JY
ICSN-RMN, Ecole Polytechnique, 91128 Palaiseau.
Leclaire
Bayesté
B
Sermet
Isabelle
I
Lenoir
Gérard
G
D018528:ATP-Binding Cassette Transporters / Q000378:metabolism; D003078:Colchicine / Q000494:pharmacology / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000378:metabolism; D006074:Gout Suppressants / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans
D018528:ATP-Binding Cassette Transporters; D006074:Gout Suppressants; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D003078:Colchicine