@article{ad801dcfb2014e57bc11a07e0e62761e,
title = "Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis",
abstract = "The use of lipid nanoparticles to deliver therapeutic mRNA is an increasingly attractive gene therapy modality. Here, Robinson et al. demonstrate that lipid nanoparticle transfection of mRNA can repair the central defect in cystic fibrosis, chloride transport, both in vitro and in vivo. The authors discuss why this technology is a promising treatment option for monogenic disorders such as cystic fibrosis.",
keywords = "cystic fibrosis, gene therapy, ion transport, mRNA therapeutics, nanoparticles, nasal potential difference",
author = "Ema Robinson and MacDonald, {Kelvin D.} and Kai Slaughter and Madison McKinney and Siddharth Patel and Conroy Sun and Gaurav Sahay",
note = "Funding Information: This project was supported by funding from the Cystic Fibrosis Foundation (to G.S. and K.D.M.), OSU College of Pharmacy startup funding (to G.S.), the National Institute of Biomedical Imaging and Bioengineering ( 1R15EB021581-01 to G.S.), and the National Institute of General Medical Sciences (NIGMS) ( 1R35GM119839-01 to C.S.). We thank Ajay Sapre for assistance with collecting single-particle tracking data and Emily Pulliam for maintenance of the CFKO mouse colony.",
year = "2018",
month = aug,
day = "1",
doi = "10.1016/j.ymthe.2018.05.014",
language = "English (US)",
volume = "26",
pages = "2034--2046",
journal = "Molecular Therapy",
issn = "1525-0016",
publisher = "Nature Publishing Group",
number = "8",
}