Molecule Shows Early Potential in Treating CF Due to F508del Mutation

Molecule Shows Early Potential in Treating CF Due to F508del Mutation

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Molecule Shows Early Potential in Treating CF Due to F508del Mutation

An orally available small molecule called XL147 rescued the function of CFTR — the faulty protein in cystic fibrosis (CF) patients — in lab-grown airway cells from patients carrying F508del, the most common CF-causing mutation, a study shows. Adding XL147 to C18, a compound similar to the CFTR modulator lumacaftor (VX-809), also resulted in a significantly greater CFTR rescue relative to C18 alone, suggesting that combining CFTR modulators or correctors with small molecules that act…

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