Browsing by Title "A BAC transgene expressing human CFTR under control of its regulatory elements rescues Cftr knockout mice"

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  • A BAC transgene expressing human CFTR under control of its regulatory elements rescues Cftr knockout mice 

    Gawenis L. R.; Hodges C. A.; McHugh D. R.; Valerio D. M.; Miron A.; Cotton C. U.; Liu J.; Walker N. M.; Strubberg A. M.; Gillen A. E.; Mutolo M. J.; Kotzamanis G.; Bosch J.; Harris A.; Drumm M. L.; Clarke L. L. (Nature Publishing Group, 2019)
    Small-molecule modulators of cystic fibrosis transmembrane conductance regulator (CFTR) biology show promise in the treatment of cystic fibrosis (CF). A Cftr knockout (Cftr KO) mouse expressing mutants of human CFTR would ...