What is
Gene Therapy?
How does Gene Therapy work?
Why use Gene Therapy to treat
CF?
CF Gene Therapy - progress to date
The future of CF Gene Therapy
The scientific principle of gene therapy for CF is a sound one and the technology already exists to transfer the CF gene into the airway cells in man but there are still challenges ahead of the field. The two main challenges are getting the gene into the cells more efficiently and to make gene expression last longer. However the field is moving forward rapidly and with the development of better new viruses and liposomes
these problems are surmountable. The members of the UK Cystic Fibrosis Gene
Therapy Consortium have played a significant role in bringing the field of gene
therapy for CF to where it is today. After initial encouraging clinical trials
most research for CF gene therapy has returned to the laboratory to solve the
problems mentioned above. In the UK there are no trials ongoing at present. It
is the goal of the Consortium to work together to meet the challenges and
enhance progress to a Phase 3 (large-scale) study by 2009.
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