Measuring the potential changes in Chloride ion conductance we are able to descriminate between CF and no-CF patients.
Patients receiving the placebo control show no significant changes following treatment.
In the patients with the high dose formulation, the potential difference is restored to normal levels following treatment. In conclusion, the single-dose study focussed our minds on the feasibility of our clinical programme. We successfully addressed DNA production and regulatory issues such that 6 plasmids produced in the UK using our regulatory protocols have now been approved by the MCA for use in clincal studies. We also developed suitable end-point assays in collaboration with the other groups. Finally, the gene transfer observed in 6 out of 8 treated patients was of limited duration and showed no dose dependency.