Cystic fibrosis: therapies targeting specific gene defects.
Cystic Fibrosis (CF) is caused by a large number of mutations in the CFTR gene, leading to specific classes of protein defects. This review discusses these classes, an understanding of which has paved the way for novel treatment strategies. The progress in this field, through from basic research to, in one case, application for license, is described.
National Heart and Lung Institute, Imperial College, Manresa Road, London, SW3 6LR. r.thursfield11@imperial.ac.uk
Rebecca M
RM
Thursfield
Jane C
JC
Davies
D003550:Cystic Fibrosis / Q000235:genetics* / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000502:physiology*; D015316:Genetic Therapy; D006801:Humans; D058990:Molecular Targeted Therapy; D009154:Mutation / Q000235:genetics
D019005:Cystic Fibrosis Transmembrane Conductance Regulator