Access to this article is restricted until 12 months after publication by request of the publisher.. Restriction lift date: 2023-10-30
Development of novel therapeutics for all individuals with CF (the future goes on)
| dc.check.date | 2023-10-30 | |
| dc.check.info | Access to this article is restricted until 12 months after publication by request of the publisher. | en |
| dc.contributor.author | Amaral, Margarida D. | |
| dc.contributor.author | Harrison, Patrick T. | |
| dc.contributor.funder | Science Foundation Ireland | en |
| dc.contributor.funder | Horizon 2020 | en |
| dc.contributor.funder | Fundação para a Ciência e a Tecnologia | en |
| dc.contributor.funder | Ministério da Ciência, Tecnologia e Ensino Superior | en |
| dc.contributor.funder | Cystic Fibrosis Trust | en |
| dc.contributor.funder | Cystic Fibrosis Foundation | en |
| dc.date.accessioned | 2022-11-10T11:44:31Z | |
| dc.date.available | 2022-11-10T11:44:31Z | |
| dc.date.issued | 2022-10-30 | |
| dc.date.updated | 2022-11-10T10:17:42Z | |
| dc.description.abstract | Despite 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.sponsorship | Science 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.status | Peer reviewed | en |
| dc.description.version | Accepted Version | en |
| dc.format.mimetype | application/pdf | en |
| dc.identifier.citation | Amaral, 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.2022.10.007 | en |
| dc.identifier.doi | 10.1016/j.jcf.2022.10.007 | en |
| dc.identifier.issn | 1569-1993 | |
| dc.identifier.journaltitle | Journal of Cystic Fibrosis | en |
| dc.identifier.uri | https://hdl.handle.net/10468/13845 | |
| dc.language.iso | en | en |
| dc.publisher | Elsevier B.V. | en |
| dc.relation.project | info:eu-repo/grantAgreement/EC/H2020::RIA/755021/EU/Personalised Treatment For Cystic Fibrosis Patients With Ultra-rare CFTR Mutations (and beyond)/HIT-CF | en |
| 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.uri | https://creativecommons.org/licenses/by-nc-nd/4.0/ | en |
| dc.subject | CFTR | en |
| dc.subject | Cystic fibrosis | en |
| dc.subject | Genome editing | en |
| dc.subject | Theratypes | en |
| dc.title | Development of novel therapeutics for all individuals with CF (the future goes on) | en |
| dc.type | Article (peer-reviewed) | en |
