Gene Therapy
Why use Gene Therapy to treat CF?
Conventional treatments for CF include the use of physiotherapy, antibiotics and pancreatic supplements. Many patients require treatment four times daily, including a considerable time spent on physiotherapy. This combination of treatment has helped increase life expectancy considerably to the current median of approximately 30 years of age. Despite small further improvements, more recently it has become apparent that there is a need for a more effective and convenient therapy. The identification of the gene responsible for CF (the cystic fibrosis transmembrane conductance regulator or CFTR protein) in 1989, was responsible for the advent of potential new treatments for CF. Gene therapy, the transfer of a normal copy of the CFTR gene into the lungs of CF patients, was proposed as an attractive new option.
Go Back