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A BAC Transgene Expressing Human CFTR under Control of Its Regulatory Elements Rescues Cftr Knockout Mice

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 advance in vivo testing of new modulators. A bacterial artificial chromosome (...

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Bibliographic Details
Published in:Sci Rep
Main Authors: Gawenis, Lara R., Hodges, Craig A., McHugh, Daniel R., Valerio, Dana M., Miron, Alexander, Cotton, Calvin U., Liu, Jinghua, Walker, Nancy M., Strubberg, Ashlee M., Gillen, Austin E., Mutolo, Michael J., Kotzamanis, George, Bosch, Jürgen, Harris, Ann, Drumm, Mitchell L., Clarke, Lane L.
Format: Artigo
Language:Inglês
Published: Nature Publishing Group UK 2019
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6694137/
https://ncbi.nlm.nih.gov/pubmed/31413336
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-48105-4
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