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Novel therapeutic approaches for the management of cystic fibrosis
Cystic fibrosis (CF) is a genetic condition characterised by the build-up of thick, sticky mucus that can damage many of the body’s organs. It is a life-long disease that results in a shortened life expectancy, often due to the progression of advanced lung disease. Treatment has previously targeted...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
PAGEPress Publications, Pavia, Italy
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7706361/ https://www.ncbi.nlm.nih.gov/pubmed/33282281 http://dx.doi.org/10.4081/mrm.2020.690 |
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author | Jaques, Ryan Shakeel, Arslan Hoyle, Cameron |
author_facet | Jaques, Ryan Shakeel, Arslan Hoyle, Cameron |
author_sort | Jaques, Ryan |
collection | PubMed |
description | Cystic fibrosis (CF) is a genetic condition characterised by the build-up of thick, sticky mucus that can damage many of the body’s organs. It is a life-long disease that results in a shortened life expectancy, often due to the progression of advanced lung disease. Treatment has previously targeted the downstream symptoms such as diminished mucus clearance and recurrent infection. More recently, significant advances have been made in treating the cause of the disease by targeting the faulty gene responsible. Hope for the development of potential therapies lies with ongoing research into new pharmacological agents and gene therapy. This review gives an overview of CF, and summarises the current evidence regarding the disease management and upcoming strategies aimed at treating or potentially curing this condition. |
format | Online Article Text |
id | pubmed-7706361 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | PAGEPress Publications, Pavia, Italy |
record_format | MEDLINE/PubMed |
spelling | pubmed-77063612020-12-03 Novel therapeutic approaches for the management of cystic fibrosis Jaques, Ryan Shakeel, Arslan Hoyle, Cameron Multidiscip Respir Med Review Cystic fibrosis (CF) is a genetic condition characterised by the build-up of thick, sticky mucus that can damage many of the body’s organs. It is a life-long disease that results in a shortened life expectancy, often due to the progression of advanced lung disease. Treatment has previously targeted the downstream symptoms such as diminished mucus clearance and recurrent infection. More recently, significant advances have been made in treating the cause of the disease by targeting the faulty gene responsible. Hope for the development of potential therapies lies with ongoing research into new pharmacological agents and gene therapy. This review gives an overview of CF, and summarises the current evidence regarding the disease management and upcoming strategies aimed at treating or potentially curing this condition. PAGEPress Publications, Pavia, Italy 2020-11-26 /pmc/articles/PMC7706361/ /pubmed/33282281 http://dx.doi.org/10.4081/mrm.2020.690 Text en ©Copyright: the Author(s) http://creativecommons.org/licenses/by-nc/4.0/ This article is distributed under the terms of the Creative Commons Attribution Noncommercial License (by-nc 4.0) which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and source are credited. |
spellingShingle | Review Jaques, Ryan Shakeel, Arslan Hoyle, Cameron Novel therapeutic approaches for the management of cystic fibrosis |
title | Novel therapeutic approaches for the management of cystic fibrosis |
title_full | Novel therapeutic approaches for the management of cystic fibrosis |
title_fullStr | Novel therapeutic approaches for the management of cystic fibrosis |
title_full_unstemmed | Novel therapeutic approaches for the management of cystic fibrosis |
title_short | Novel therapeutic approaches for the management of cystic fibrosis |
title_sort | novel therapeutic approaches for the management of cystic fibrosis |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7706361/ https://www.ncbi.nlm.nih.gov/pubmed/33282281 http://dx.doi.org/10.4081/mrm.2020.690 |
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