Related Experiment Video

Updated: Aug 11, 2026

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein
09:59

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein

Published on: March 9, 2015

On the discovery and development of CFTR chloride channel activators

Frédéric Becq1

  • 1Institut de Physiologie et Biologie Cellulaires CNRS UMR 6187, Université de Poitiers, 40 Avenue du Recteur Pineau 86022 Poitiers, France. frederic.becq@univ-poitiers.fr

Current Pharmaceutical Design
|February 14, 2006
PubMed

Insights

Researchers are developing new drugs to treat cystic fibrosis (CF) by targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein. High-throughput screening is identifying novel molecules to restore CFTR function and potentially cure CF.

Area of Science:

  • Cellular Biology
  • Molecular Biology
  • Medical Genetics

Background:

  • Chloride channels are crucial for cellular homeostasis.
  • Mutations in ion channel genes, including CFTR, cause hereditary diseases like cystic fibrosis.
  • The CFTR protein functions as an ATP-gated and phosphorylation-regulated chloride channel.

Purpose of the Study:

  • To review the evolution of CFTR pharmacology.
  • To highlight the development of high-throughput screening assays for identifying CFTR modulators.
  • To discuss the potential of small molecules as therapeutic agents for cystic fibrosis.

Main Methods:

  • Literature review focusing on CFTR pharmacology.
  • Analysis of high-throughput screening assay development over the last five years.
  • Identification of novel small molecules modulating CFTR function.

Main Results:

  • Significant advancements in understanding CFTR's physiological role.
  • Development of robust screening assays for identifying CFTR modulators.
  • Discovery of potential therapeutic agents for cystic fibrosis.

Conclusions:

  • Pharmacological modulation of CFTR offers a promising therapeutic strategy for cystic fibrosis.
  • Novel small molecules identified through screening may restore defective CFTR transport and regulatory functions.
  • Continued research in CFTR pharmacology is essential for developing effective treatments for CF.

More Related Videos

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients07:04

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

A Fluorescence-Based Assay of Membrane Potential for High-Throughput Functional Study of Two Endogenous Ion Channels in Two Epithelial Cell Lines06:59

A Fluorescence-Based Assay of Membrane Potential for High-Throughput Functional Study of Two Endogenous Ion Channels in Two Epithelial Cell Lines

Published on: June 22, 2022

Related Concept Videos

Cystic Fibrosis: Pathogenesis01:23

Cystic Fibrosis: Pathogenesis

Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Patch Clamp01:18

Patch Clamp

Many fundamental cell functions such as muscle contraction and nerve transmission rely on the electrical signals produced by the movement of positively and negatively charged ions across the cell membrane. One competent method to record current flowing across the whole cell or single ion channel is the patch-clamp technique.
In this method, a glass micropipette containing electrolyte solution is tightly sealed against a small portion of the cell membrane. As a result, a patch of the cell...
Drug Discovery: Overview01:26

Drug Discovery: Overview

Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
Transcellular Transport of Solutes01:23

Transcellular Transport of Solutes

Transcellular transport of solutes is the movement of substances like monosaccharides and amino acids through polarized cells. This transport mechanism is primarily seen in epithelial and endothelial cells aided by membrane transport proteins such as channels and transporters. The tight junctions between these cells confine the membrane proteins to the two sides of the cell. The epithelial cells have distinct apical and basolateral domains. In contrast, the endothelial cells show the luminal...
Facilitated Transport01:19

Facilitated Transport

The chemical and physical properties of plasma membranes cause them to be selectively permeable. Since plasma membranes have both hydrophobic and hydrophilic regions, substances need to be able to transverse both regions. The hydrophobic area of membranes repels substances such as charged ions. Therefore, such substances need special membrane proteins to cross a membrane successfully. In  facilitated transport, also known as facilitated diffusion, molecules and ions travel across a membrane via...