Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia

Cystic fibrosis: toward personalized therapies A new method for evaluating drug responses in patient-derived respiratory tissue promises to help determine the best treatment for each patient with cystic fibrosis (CF). CF patients are highly susceptible to lung infections due to the build-up of thick...

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Autores principales:	Saumel Ahmadi, Zoltan Bozoky, Michelle Di Paola, Sunny Xia, Canhui Li, Amy P. Wong, Leigh Wellhauser, Steven V. Molinski, Wan Ip, Hong Ouyang, Julie Avolio, Julie D. Forman-Kay, Felix Ratjen, Jeremy A. Hirota, Johanna Rommens, Janet Rossant, Tanja Gonska, Theo J. Moraes, Christine E. Bear
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2017
Materias:	Medicine R Genetics QH426-470
Acceso en línea:	https://doaj.org/article/7ad6ed4d84964bd982f178ae435366d1
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spelling	oai:doaj.org-article:7ad6ed4d84964bd982f178ae435366d12021-12-02T15:10:32ZPhenotypic profiling of CFTR modulators in patient-derived respiratory epithelia10.1038/s41525-017-0015-62056-7944https://doaj.org/article/7ad6ed4d84964bd982f178ae435366d12017-04-01T00:00:00Zhttps://doi.org/10.1038/s41525-017-0015-6https://doaj.org/toc/2056-7944Cystic fibrosis: toward personalized therapies A new method for evaluating drug responses in patient-derived respiratory tissue promises to help determine the best treatment for each patient with cystic fibrosis (CF). CF patients are highly susceptible to lung infections due to the build-up of thick mucus in the airways. Over 2000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been identified in patients with CF, which partly explains their varied response to treatment. Saumel Ahmadi, Christine E. Bear, and colleagues at the Hospital for Sick Children in Toronto developed a fluorescence-based method for measuring improvements in mutant CFTR function in patient-derived nasal and induced pluripotent stem cell-derived lung tissue. This method enables comparison of approved and investigational drugs on airway cells from each individual patient and in the longer term will accelerate the development of personalized therapeutic strategies.Saumel AhmadiZoltan BozokyMichelle Di PaolaSunny XiaCanhui LiAmy P. WongLeigh WellhauserSteven V. MolinskiWan IpHong OuyangJulie AvolioJulie D. Forman-KayFelix RatjenJeremy A. HirotaJohanna RommensJanet RossantTanja GonskaTheo J. MoraesChristine E. BearNature PortfolioarticleMedicineRGeneticsQH426-470ENnpj Genomic Medicine, Vol 2, Iss 1, Pp 1-10 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Genetics QH426-470
spellingShingle	Medicine R Genetics QH426-470 Saumel Ahmadi Zoltan Bozoky Michelle Di Paola Sunny Xia Canhui Li Amy P. Wong Leigh Wellhauser Steven V. Molinski Wan Ip Hong Ouyang Julie Avolio Julie D. Forman-Kay Felix Ratjen Jeremy A. Hirota Johanna Rommens Janet Rossant Tanja Gonska Theo J. Moraes Christine E. Bear Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
description	Cystic fibrosis: toward personalized therapies A new method for evaluating drug responses in patient-derived respiratory tissue promises to help determine the best treatment for each patient with cystic fibrosis (CF). CF patients are highly susceptible to lung infections due to the build-up of thick mucus in the airways. Over 2000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been identified in patients with CF, which partly explains their varied response to treatment. Saumel Ahmadi, Christine E. Bear, and colleagues at the Hospital for Sick Children in Toronto developed a fluorescence-based method for measuring improvements in mutant CFTR function in patient-derived nasal and induced pluripotent stem cell-derived lung tissue. This method enables comparison of approved and investigational drugs on airway cells from each individual patient and in the longer term will accelerate the development of personalized therapeutic strategies.
format	article
author	Saumel Ahmadi Zoltan Bozoky Michelle Di Paola Sunny Xia Canhui Li Amy P. Wong Leigh Wellhauser Steven V. Molinski Wan Ip Hong Ouyang Julie Avolio Julie D. Forman-Kay Felix Ratjen Jeremy A. Hirota Johanna Rommens Janet Rossant Tanja Gonska Theo J. Moraes Christine E. Bear
author_facet	Saumel Ahmadi Zoltan Bozoky Michelle Di Paola Sunny Xia Canhui Li Amy P. Wong Leigh Wellhauser Steven V. Molinski Wan Ip Hong Ouyang Julie Avolio Julie D. Forman-Kay Felix Ratjen Jeremy A. Hirota Johanna Rommens Janet Rossant Tanja Gonska Theo J. Moraes Christine E. Bear
author_sort	Saumel Ahmadi
title	Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
title_short	Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
title_full	Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
title_fullStr	Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
title_full_unstemmed	Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia
title_sort	phenotypic profiling of cftr modulators in patient-derived respiratory epithelia
publisher	Nature Portfolio
publishDate	2017
url	https://doaj.org/article/7ad6ed4d84964bd982f178ae435366d1
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Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia

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