Development of novel therapeutics for all individuals with CF (the future goes on)

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dc.check.date2023-10-30
dc.check.infoAccess to this article is restricted until 12 months after publication by request of the publisher.en
dc.contributor.authorAmaral, Margarida D.
dc.contributor.authorHarrison, Patrick T.
dc.contributor.funderScience Foundation Irelanden
dc.contributor.funderHorizon 2020en
dc.contributor.funderFundação para a Ciência e a Tecnologiaen
dc.contributor.funderMinistério da Ciência, Tecnologia e Ensino Superioren
dc.contributor.funderCystic Fibrosis Trusten
dc.contributor.funderCystic Fibrosis Foundationen
dc.date.accessioned2022-11-10T11:44:31Z
dc.date.available2022-11-10T11:44:31Z
dc.date.issued2022-10-30
dc.date.updated2022-11-10T10:17:42Z
dc.description.abstractDespite the major advances and successes in finding and establishing new treatments that tackle the basic defect in Cystic Fibrosis (CF), there is still an unmet need to bring these potentially curative therapies to all individuals with CF. Here, we review aspects of what is still missing to treat all individuals with CF by such approaches. On the one hand, we discuss novel holistic (high-throughput) approaches to elucidate mechanistic defects caused by distinct classes of mutations to identify novel drug targets. On the other hand, we examine therapeutic approaches to correct the gene in its own environment, i.e., in the genome.en
dc.description.sponsorshipScience Foundation Ireland (20-FFP-P-8661); Fundação para a Ciência e a Tecnologia / Ministério da Ciência, Tecnologia e Ensino Superior (UIDB/04046/2020; UIDP/04046/2020); Cystic Fibrosis Trust (SRC 013; SRC020); Cystic Fibrosis Foundation (AMARAL19G0; ALLEN20G0; HARRIS20G0; HARRISP21G0)en
dc.description.statusPeer revieweden
dc.description.versionAccepted Versionen
dc.format.mimetypeapplication/pdfen
dc.identifier.citationAmaral, M. D. and Harrison, P. T. (2022) 'Development of novel therapeutics for all individuals with CF (the future goes on)', Journal of Cystic Fibrosis. doi: 10.1016/j.jcf.2022.10.007en
dc.identifier.doi10.1016/j.jcf.2022.10.007en
dc.identifier.issn1569-1993
dc.identifier.journaltitleJournal of Cystic Fibrosisen
dc.identifier.urihttps://hdl.handle.net/10468/13845
dc.language.isoenen
dc.publisherElsevier B.V.en
dc.relation.projectinfo:eu-repo/grantAgreement/EC/H2020::RIA/755021/EU/Personalised Treatment For Cystic Fibrosis Patients With Ultra-rare CFTR Mutations (and beyond)/HIT-CFen
dc.rights© 2022, European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved. This manuscript version is made available under the CC BY-NC-ND 4.0 license.en
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/4.0/en
dc.subjectCFTRen
dc.subjectCystic fibrosisen
dc.subjectGenome editingen
dc.subjectTheratypesen
dc.titleDevelopment of novel therapeutics for all individuals with CF (the future goes on)en
dc.typeArticle (peer-reviewed)en
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