Research updates
3 - Cystic fibrosis: the quest for a cure page 7
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4. autious optimism Link to the Medical Research Council web site
Looking to the future
More than 100 patients with CF have now taken part in clinical trials in Europe and the USA. Both viral and liposome vectors have shown promise, but neither system is yet efficient enough to provide a treatment. The results are exciting, but it is early days for gene therapy and research continues apace.
The ultimate goal of CF gene therapy is to provide a life-long cure for sufferers. Scientists world-wide are working together to achieve this goal. In the words of MRC scientist David Porteous, "there is little doubt that, in time, gene therapy will allow the sticky mucus to thin and flow freely, so flushing out infection and keeping the lung 'in the pink' ".
Question 5
From the description above, comment on the design of the US human trial. How reliable are the results? How would you improve the experimental design?

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