Description

• Title:
  Modulators of CFTR protein function in the treatment of cystic fibrosis - a literature review
• Creator:
  Chojęta, Dariusz;
  Mierzwa, Monika;
  Wróblewski, Hubert;
  Zimna, Aleksandra;
  Zygmunt, Ewelina;
  Kozłowska, Anna;
  Łysiak, Katarzyna
• Description:
  Cystic fibrosis (CF) is a multi-system genetic disease with an autosomal recessive inheritance mechanism. The breakthroughs in the therapy of patients with CF turned out to be modulators of CFTR protein function. These small-molecule substances, influencing the basic pathogenetic defect, became a model example of precise targeted therapy, thus showing a significant advantage over symptomatic treatment. For modulators of CFTR protein function, numerous therapeutic benefits have been demonstrated with an acceptable safety profile - improved lung function and reduced frequency of pulmonary exacerbations, reduced hospitalization rate, and disease-related improvement in quality of life. The use of these drugs changes the perception of CF from a fatal pediatric disease significantly shortening the life expectancy of patients into a chronic disease in young adults. The subject of further research interest is the development of alternative therapeutic strategies based on new modulators and gene therapy. The publication is a review of the literature and summarizes the current scientific reports on the effectiveness of the use of modulators of CFTR protein function in the treatment of cystic fibrosis.
• Object availability:
  free access
• Rights:
  Prawa autorskie (c) 2022 Dariusz Chojęta, Monika Mierzwa, Hubert Wróblewski, Aleksandra Zimna, Ewelina Zygmunt, Anna Kozłowska, Katarzyna Łysiak;
  http://creativecommons.org/licenses/by-nc-sa/4.0
• Date:
  2022.08.09
• Type:
  info:eu-repo/semantics/article;
  info:eu-repo/semantics/publishedVersion
• Language:
  English
• Source:
  Journal of Education, Health and Sport; Vol. 12 No. 8 (2022); 358-365;
  Journal of Education, Health and Sport; Tom 12 Nr 8 (2022); 358-365;
  2391-8306
• Format:
  application/pdf
• Publisher:
  Nicolaus Copernicus University, Toruń, Poland
• Relation: https://apcz.umk.pl/JEHS/article/view/39321/32930
• Subject:
  cystic fibrosis;
  modulators of CFTR protein function;
  targeted therapy;
  gene therapy
• Identifier: https://apcz.umk.pl/JEHS/article/view/39321
• Data provider:
  Akademicka Platforma Czasopism
• Can I use it?:
  seek permission
• Type:
  text