Therapeutic strategies for treatment of CF based on knowledge of CFTR.
CF is one of the first diseases where it is now possible to take an entirely "bottom-up" approach and attempt to develop molecular therapeutics based on fundamental properties of the gene and gene product which cause the disease. As I have tried to illustrate this is a task of enormous magnitude and although significant progress is being made, it is reasonable to expect that considerable time may be required for a satisfactory outcome to be achieved.
Riordan
J R
JR
Department of Research, Mayo Clinic Scottsdale, Arizona 85259, USA.
D002648:Child; D003550:Cystic Fibrosis / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000627:therapeutic use*; D015316:Genetic Therapy; D006801:Humans
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator