Research ArticleCystic Fibrosis

In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis

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Science Translational Medicine  27 Mar 2019:
Vol. 11, Issue 485, eaau7531
DOI: 10.1126/scitranslmed.aau7531

Tackling cystic fibrosis in the womb

Cystic fibrosis (CF) is a multiorgan disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Abnormalities are observed early during development. Sun et al. reasoned that early intervention using the approved drug VX-770 (ivacaftor), a CFTR modulator, could prevent the development of such abnormalities. The authors tested the effect of in utero and early postnatal VX-770 administration in a ferret model of CF. Lung, gastrointestinal, pancreatic, and male reproductive pathologies were partially prevented in ferrets treated prior to and following birth. The results suggest that CFTR plays a critical role in early organ development and that prenatal and postnatal treatment might prevent developmental pathologies and onset of disease.

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