Cystic fibrosis manifests when the is either not made correctly, or absent. Researchers say there are numerous subclasses of molecular defects in thecystic fibrosis transmembrane conductance regulator (CFTR) protein, which makes personalized approaches complex, reports Science Magazine.
For many years, a conceptual approach to CF intervention has pursued tailored small molecules (modulators) designed to rescue specific CFTR defects. Personalized treatment strategies based on this early annotation have led to impressive therapeutic progress.
It was originally anticipated that well-established disease subclasses would serve as an organizing principle for precision CF treatments and help identify specific compounds for targeting CFTR mutants in a mechanism-directed manner. However, it has become increasingly clear that most CFTR variants result in not just one, but numerous subclasses of molecular defects in the CFTR protein, which makes personalized approaches complex.