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In utero and postnatal ivacaftor/lumacaftor therapy rescues multiorgan disease in CFTR-F508del ferrets.
Evans IA, Sun X, Liang B, Vegter AR, Guo L, Lynch TJ, Zhang Y, Zhang Y, Yi Y, Yang Y, Feng Z, Park SY, Shonka A, McCumber H, Qi L, Wu P, Liu G, Lacina A, Wang K, Gibson-Corley KN, Meyerholz DK, Limoli DH, Rosen BH, Yan Z, Bartels DJ, Engelhardt JF. Evans IA, et al. Among authors: vegter ar. JCI Insight. 2024 Apr 22;9(8):e157229. doi: 10.1172/jci.insight.157229. JCI Insight. 2024. PMID: 38646935 Free PMC article.
Ferret models of alpha-1 antitrypsin deficiency develop lung and liver disease.
He N, Liu X, Vegter AR, Evans TIA, Gray JS, Guo J, Moll SR, Guo LJ, Luo M, Ma N, Sun X, Liang B, Yan Z, Feng Z, Qi L, Joshi AS, Shahin W, Yi Y, Gibson-Corley KN, Hoffman EA, Wang K, Mueller C, Engelhardt JF, Rosen BH. He N, et al. Among authors: vegter ar. JCI Insight. 2022 Mar 8;7(5):e143004. doi: 10.1172/jci.insight.143004. JCI Insight. 2022. PMID: 35104244 Free PMC article.