@article{0755e5e73d0a4882a906a4389ef51e70,
title = "Translational research to enable personalized treatment of cystic fibrosis",
abstract = "Translational research efforts in cystic fibrosis (CF) aim to develop therapies for all subjects with CF. To reach this goal new therapies need to be developed that target multiple aspects of the disease. To enable individuals to benefit maximally from these treatments will require improved methods to tailor these therapies specifically to individuals who suffer from CF. This report highlights current examples of translational CF research efforts to reach this goal. The use of intestinal organoids and genetics to better understand individual assessment of CFTR modulator treatment effects to ultimately enable a better personalized treatment for CF subjects will be discussed. In addition, development of viral vectors and non-viral synthetic nanoparticles for delivery of mRNA, sgRNA and DNA will be highlighted. New approaches to restore function of CFTR with early premature termination codons using nanoparticle delivery of suppressor tRNAs and new insights into mechanisms of airway epithelial repair will be reviewed as well. The state-of-the-art approaches that are discussed in this review demonstrate significant progress towards the development of optimal individual therapies for CF patients, but also reveal that remaining challenges still lie ahead.",
keywords = "CFTR modulator, Cystic fibrosis, Gene therapy, Nanoparticles, Personalized medicine, Translational research, tRNA",
author = "Hagemeijer, {Marne C.} and Siegwart, {Daniel J.} and Strug, {Lisa J.} and Liudmila Cebotaru and Torres, {Michael J.} and Aderonke Sofoluwe and Beekman, {Jeffrey M.}",
note = "Funding Information: JMB was sponsored by the Dutch CF society, ZonMW ( 40-00812-98-14103 ), H2020 ( 633545 ) and the Wilhelmina Children's hospital . AS has been funded by Swiss National Science Foundation ( SNF ) ( 310030_153342 ), Swiss CF foundation and ABCF Mucoviscidose . DJS acknowledges financial support from the Cancer Prevention and Research Institute of Texas ( R1212 ), the Welch Foundation ( I-1855 ), the American Cancer Society ( RSG-17-012-01 ), and the Mary Kay Foundation ( 049-15 ). Work by MJT supported by CFFT, Inc. ( RECODE17X0-SC ) and ReCode Therapeutics, Inc. LJS has been funded by the Canadian Institutes of Health Research (CIHR; MOP-258916 ); the Natural Sciences and Engineering Research Council of Canada (NSERC; 371399-2009 ); and Cystic Fibrosis Canada # 2626 . LC was funded by the National Institutes of Health R01 HL 122267 and by the Cystic Fibrosis Foundation (USA). Funding Information: JMB was sponsored by the Dutch CF society, ZonMW (40-00812-98-14103), H2020 (633545) and the Wilhelmina Children's hospital. AS has been funded by Swiss National Science Foundation (SNF) (310030_153342), Swiss CF foundation and ABCF Mucoviscidose. DJS acknowledges financial support from the Cancer Prevention and Research Institute of Texas (R1212), the Welch Foundation (I-1855), the American Cancer Society (RSG-17-012-01), and the Mary Kay Foundation (049-15). Work by MJT supported by CFFT, Inc. (RECODE17X0-SC) and ReCode Therapeutics, Inc. LJS has been funded by the Canadian Institutes of Health Research (CIHR; MOP-258916); the Natural Sciences and Engineering Research Council of Canada (NSERC; 371399-2009); and Cystic Fibrosis Canada # 2626. LC was funded by the National Institutes of Health R01 HL 122267 and by the Cystic Fibrosis Foundation (USA). Publisher Copyright: {\textcopyright} 2017 European Cystic Fibrosis Society",
year = "2018",
month = mar,
doi = "10.1016/j.jcf.2017.10.017",
language = "English (US)",
volume = "17",
pages = "S46--S51",
journal = "Journal of Cystic Fibrosis",
issn = "1569-1993",
publisher = "Elsevier",
number = "2",
}