First real-world study of fetal therapy with CFTR modulators in cystic fibrosis: Report from the MODUL-CF study

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

Xingshen Sun, Yaling Yi, Ziying Yan et al. · Science Translational Medicine · 2019 · 185 citations · Full text

Life Sciences, Genetics, Physiology +17

Expression of the cystic fibrosis gene in human foetal tissues

A. E. O. Trezise, Julie A. Chambers, Catherine J. Wardle et al. · Human Molecular Genetics · 1993 · 159 citations

Life Sciences, Genetics, Molecular Genetics +6

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

David A. Stoltz, Tatiana Rokhlina, Sarah E. Ernst et al. · Journal of Clinical Investigation · 2013 · 122 citations · Full text

Life Sciences, Molecular Biology, Physiology +16

Normal pancreatic function and false-negative CF newborn screen in a child born to a mother taking CFTR modulator therapy during pregnancy

Christopher N. Fortner, Julie M. Seguin, Denise M. Kay · Journal of Cystic Fibrosis · 2021 · 110 citations

Medicine, Child Health, Pediatrics +14

A case report of CFTR modulator administration via carrier mother to treat meconium ileus in a F508del homozygous fetus

Sylvia Szentpetery, Kimberly Foil, Sara J. Hendrix et al. · Journal of Cystic Fibrosis · 2022 · 100 citations

Medicine, Genetics, Obstetrics +10