Cystic fibrosis remains the most common fatal genetic disease in Europe. Advanced medical treatment has raised the average life span to 30 years. Treatments so far manage acute symptoms and slow the disease progression. Gene therapy may open the door for further progress.
Disease: Cystic Fibrosis
Last update: June 2008
Intro
FAQ: What does the future hold?
Research is underway on an inhaled form of a particular protein which is lacking in the lungs of CF patients. Research also continues into an alternative to existing pancreatic enzyme preparations and, into a small 'repair' molecule which binds to the defective CFTR protein and improves its function.
Pseudomonas aeruginosa is a bacterium which commonly infects the lungs of patients with CF. It often develops resistance to many antibiotics. An inhaled antibiotic to combat it is now available. A major step forward has been the publication of the complete genetic structure of Pseudomonas. This is an important tool in the development of new therapies against it. A new vaccine against Pseudomonas is also being developed.
Gene therapy, where a normal version of the CFTR gene is inserted into the lungs and digestive system, is a feasible approach but much more research is needed to turn it into a viable therapy.
Pseudomonas aeruginosa is a bacterium which commonly infects the lungs of patients with CF. It often develops resistance to many antibiotics. An inhaled antibiotic to combat it is now available. A major step forward has been the publication of the complete genetic structure of Pseudomonas. This is an important tool in the development of new therapies against it. A new vaccine against Pseudomonas is also being developed.
Gene therapy, where a normal version of the CFTR gene is inserted into the lungs and digestive system, is a feasible approach but much more research is needed to turn it into a viable therapy.