New Therapeutic Approaches in Cystic Fibrosis

Journal Title: Turkish Journal of Pharmaceutical Sciences - Year 2020, Vol 17, Issue 6

Abstract

Cystic fibrosis (CF) is a hereditary, multisystemic disease caused by different mutations in the CFTR gene encoding CF transmembrane conductance regulator. CF is mainly characterized by pulmonary dysfunction as a result of deterioration in the mucociliary clearance and anion transport of airways. Mortality is mostly caused by bronchiectasis, bronchiole obstruction, and progressive respiratory dysfunction in the early years of life. Over the last decade, new therapeutic strategies rather than symptomatic treatment have been proposed, such as the small molecule approach, ion channel therapy, and pulmonary gene therapy. Due to considerable progress in the treatment options, CF has become an adult disease rather than a pediatric disease in recent years. Pulmonary gene therapy has gained special attention due to its mutation type independent aspect, therefore being applicable to all CF patients. On the other hand, the major obstacle for CF treatment is to predict the drug response of patients due to genetic complexity and heterogeneity. The advancement of 3D culture systems has made it possible to extrapolate the disease modeling and individual drug response in vitro by producing mini adult organs called “organoids” obtained from rectal cell biopsies. In this review, we summarize the advances in the novel therapeutic approaches, clinical interventions, and precision medicine concept for CF.

Authors and Affiliations

Dolunay Merve FAKIOĞLU, Beril ALTUN

Keywords

Related Articles

In Vitro Macrophage Nitric Oxide and Interleukin-1 Beta Suppression by Moringa peregrina Seed

Objectives: Moringa peregrina has long been used in folk medicine to treat diseases including fever, headache, burns, constipation, gut pains, and inflammation. Nitric oxide (NO) and interleukin-1β (IL-1β) play an import...

Subchronic Toxicity Assessment of Orally Administered Methanol (70%) Seed Extract of Abrus precatorius L. in Wistar Albino Rats

Objectives: Abrus precatorius L. is a famous medicinal plant of the family Fabaceae and is widely used in traditional medicine for the treatment of various ailments. However, there are limited toxicological data availabl...

Evaluation of the Methylation and Acetylation Profiles of Dinitroaniline Herbicides and Resveratrol on the V79 Cell Line

Objectives: Herbicides are among the most widely used pesticide compounds for plant growth control worldwide. Risk assessment of the dinitroaniline-derived herbicides pendimethalin and trifluralin is important for foodbo...

A Novel Genotyping Method for Detection of the Muscarinic Receptor M1 Gene rs2067477 Polymorphism and Its Genotype/Allele Frequencies in a Turkish Population

Objectives: Gene variation in the cholinergic muscarinic receptor 1 (CHRM1) has potential to become a candidate biomarker in the development of several disorders as well as drug response. In this study, a novel polymeras...

The In Vivo Antinociceptive and Antiinflammatory Effects of Verbascum exuberans Hub.-Mor.

Objectives: Safe and effective drugs are still lacking for many pain therapies. In recent years, growing interest has been devoted thus on herbal drugs as an option to identify new pain killers. Based on this, extensive...

Download PDF file
  • EP ID EP691702
  • DOI 10.4274/tjps.galenos.2020.76401
  • Views 130
  • Downloads 0

How To Cite

Dolunay Merve FAKIOĞLU, Beril ALTUN (2020). New Therapeutic Approaches in Cystic Fibrosis. Turkish Journal of Pharmaceutical Sciences, 17(6), -. https://europub.co.uk/articles/-A-691702