Gene Therapy
Why use Gene Therapy to treat CF?
CF Gene Therapy - progress to date
Only a year after the gene for CF was identified scientists showed in cells in the laboratory that it was possible to correct the CF defect by adding a healthy copy of the gene. These findings have been consistently repeated by other groups using a variety of models and gene transfer agents. Studies in humans followed quickly. Gene therapy for CF has been tested in humans using both viruses and liposomes. Five of the
liposome trials were undertaken by members of the UK CF Gene Therapy Consortium. These early studies were concerned mainly with safety issues. The amount of gene transfer achieved is similar for both systems and is probably still too small to have any real therapeutic benefit - although it is important to note that none of these trials actually measured therapeutic
benefit. Also at present the effect seen (gene expression) only lasts
for a few days.
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