Why wasn’t the DF508 mutation chosen for the VX-770 clinical trial?
Because G551D is at the cell’s surface, it is the obvious choice to study VX-770. Most DF508 protein is not at the cell surface and therefore it is unclear whether the patients with this mutation will benefit, but it is possible. These patients may need to have the location of their CFTR protein corrected to achieve the most benefit from VX-770.
