Article
In utero and postnatal ivacaftor/lumacaftor therapy rescues multiorgan disease in CFTR-F508del ferrets.
JCI insight - 22 Apr 2024
Evans Idil Apak, Sun Xingshen, Liang Bo, Vegter Amber R, Guo Lydia, Lynch Thomas J, Zhang Yan, Zhang Yulong, Yi Yaling, Yang Yu, Feng Zehua, Park Soo Yeun, Shonka Amanita, McCumber Hannah, Qi Lisi, Wu Peipei, Liu Guangming, Lacina Allison, Wang Kai, Gibson-Corley Katherine N, Meyerholz David K, Limoli Dominique H, Rosen Bradley H, Yan Ziying, Bartels Douglas J, Engelhardt John F
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, with F508del being the most prevalent mutation. The combination of CFTR modulators (potentiator and correctors) has provided benefit to CF patients carrying the F508del mutation; however, the safety and effectiveness of in utero combination modulator therapy remains unclear. We created a F508del ferret model to...
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