Cystic fibrosis (CF) is a devastating disease, graphically described from the patient perspective as drowning from the inside. It is an inherited disorder that causes organ damage throughout the body, with particularly severe damage to the lungs and digestive system, impairing normal growth and development.
In people with CF, a defective gene causes secretions in the lung and digestive tract to become sticky and thick. This leads to ducts, tubes, and passages becoming blocked, causing tissue damage and increasing the risk of infections, especially in the lungs.
Cystic Fibrosis is a channelopathy caused by mutations of the CFTR gene that lead to defects in CFTR chloride ion channel function, resulting in dysregulation of epithelial fluid transport in the lung. This dysregulation leads to mucus becoming thick and sticky. The role of mucus is now compromised; it fails to clear bacteria and other foreign matter, leading to infection and inflammation, and can ‘plug’ the airways, further compromising lung function.
CFTR is not the only chloride ion channel present in the airway epithelium – there is also a calcium-activated chloride conductance channel (CaCC). Although the presence of this channel has been known for decades, its molecular identity was only elucidated as TMEM16A (sometimes referred to as Anoctamin1 (Ano1)), in 2008.
Below see selected publications, posters and reports on relevant ion channel targets performed on QPatch or Qube.
- View TMEM16A on Qube 384 Year: 2017
- View TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis Year: 2020
- View Development of a QPatch-Automated Electrophysiology Assay for Identifying TMEM16A Small-Molecule Inhibitors Year: 2020
- View Development of Automated Patch Clamp Technique to Investigate CFTR Chloride Channel Function Year: 2017
- View Mashiningan improves opioid-induced constipation in rats by activating cystic fibrosis transmembrane conductance regulator chloride channel Year: 2017
- View Drug Repurposing: The Anthelmintics Niclosamide and Nitazoxanide are Potent TMEM16A Antagonists that Fully Bronchodilate Airway Year: 2018
- View Novel bacterial topoisomerase inhibitors derived from isomannide Year: 2020
- View Automated Patch Clamp in Cystic Fibrosis Drug Discovery Year: 2021