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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Udgivet i: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
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2019
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Article
Online adgang: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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