CF gene therapy research
Exciting new research recently made headlines suggesting trials of gene therapy offered new hope to cystic fibrosis sufferers.
However, an analysis by NHS Choices has urged caution.
It was shown to reverse the condition in some cases. While the findings are encouraging, they also have limitations.
The trial was relatively small, recruiting just 140 patients. This is normal for a phase II trial, but large clinical trials are needed to fully assess the effects and safety of this treatment in development. The therapy was able to lessen some of the lung deterioration, but not in all. Nonetheless, this gives the researchers hope and scope to work out how to improve including optimising the dose.
Health & Living