Novel therapeutic approaches for the management of cystic fibrosis

  • Ryan Jaques | hyrj5@hyms.ac.uk Centre for Atherothrombosis and Metabolic Disease, Hull York Medical School, University of Hull, United Kingdom. https://orcid.org/0000-0002-3944-195X
  • Arslan Shakeel Centre for Atherothrombosis and Metabolic Disease, Hull York Medical School, University of Hull, United Kingdom.
  • Cameron Hoyle Centre for Atherothrombosis and Metabolic Disease, Hull York Medical School, University of Hull, United Kingdom. https://orcid.org/0000-0003-2422-0932

Abstract

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.

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Published
2020-11-26
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Issue
Section
Reviews
Keywords:
Cystic fibrosis, CFTR, treatment, infection, gene therapy
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How to Cite
Jaques, R., Shakeel, A., & Hoyle, C. (2020). Novel therapeutic approaches for the management of cystic fibrosis. Multidisciplinary Respiratory Medicine, 15. https://doi.org/10.4081/mrm.2020.690