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Current Treatment Options for Cystic Fibrosis-Related Liver Disease
Cystic Fibrosis-related liver disease (CFLD) has become a leading cause of morbidity and mortality in patients with Cystic Fibrosis (CF), and affects children and adults. The understanding of the pathogenesis of CFLD is key in order to develop efficacious treatments. However, it remains complex, and...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2020
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7696864/ https://www.ncbi.nlm.nih.gov/pubmed/33202578 http://dx.doi.org/10.3390/ijms21228586 |
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author | Staufer, Katharina |
author_facet | Staufer, Katharina |
author_sort | Staufer, Katharina |
collection | PubMed |
description | Cystic Fibrosis-related liver disease (CFLD) has become a leading cause of morbidity and mortality in patients with Cystic Fibrosis (CF), and affects children and adults. The understanding of the pathogenesis of CFLD is key in order to develop efficacious treatments. However, it remains complex, and has not been clarified to the last. The search for a drug might be additionally complicated due to the diverse clinical picture and lack of a unified definition of CFLD. Although ursodeoxycholic acid has been used for decades, its efficacy in CFLD is controversial, and the potential of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators and targeted gene therapy in CFLD needs to be defined in the near future. This review focuses on the current knowledge on treatment strategies for CFLD based on pathomechanistic viewpoints. |
format | Online Article Text |
id | pubmed-7696864 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-76968642020-11-29 Current Treatment Options for Cystic Fibrosis-Related Liver Disease Staufer, Katharina Int J Mol Sci Review Cystic Fibrosis-related liver disease (CFLD) has become a leading cause of morbidity and mortality in patients with Cystic Fibrosis (CF), and affects children and adults. The understanding of the pathogenesis of CFLD is key in order to develop efficacious treatments. However, it remains complex, and has not been clarified to the last. The search for a drug might be additionally complicated due to the diverse clinical picture and lack of a unified definition of CFLD. Although ursodeoxycholic acid has been used for decades, its efficacy in CFLD is controversial, and the potential of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators and targeted gene therapy in CFLD needs to be defined in the near future. This review focuses on the current knowledge on treatment strategies for CFLD based on pathomechanistic viewpoints. MDPI 2020-11-14 /pmc/articles/PMC7696864/ /pubmed/33202578 http://dx.doi.org/10.3390/ijms21228586 Text en © 2020 by the author. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Staufer, Katharina Current Treatment Options for Cystic Fibrosis-Related Liver Disease |
title | Current Treatment Options for Cystic Fibrosis-Related Liver Disease |
title_full | Current Treatment Options for Cystic Fibrosis-Related Liver Disease |
title_fullStr | Current Treatment Options for Cystic Fibrosis-Related Liver Disease |
title_full_unstemmed | Current Treatment Options for Cystic Fibrosis-Related Liver Disease |
title_short | Current Treatment Options for Cystic Fibrosis-Related Liver Disease |
title_sort | current treatment options for cystic fibrosis-related liver disease |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7696864/ https://www.ncbi.nlm.nih.gov/pubmed/33202578 http://dx.doi.org/10.3390/ijms21228586 |
work_keys_str_mv | AT stauferkatharina currenttreatmentoptionsforcysticfibrosisrelatedliverdisease |