Cystic Fibrosis Transmembrane Conductance Regulator Related Drug Discovery Products

The cystic fibrosis transmembrane conductance regulator (CFTR) epitomizes a protein of paramount importance in sustaining the delicate balance of salt and water within myriad tissues dispersed throughout the organism. Upon encountering compromised or non-existent functionality of the CFTR protein, stemming from genetic aberrations, the onset of cystic fibrosis, a hereditary malady, may ensue. This disorder precipitates grave repercussions for the respiratory, digestive, and reproductive systems, highlighting the indispensable role that the CFTR protein fulfills in maintaining homeostasis and ensuring the proper functioning of the body's vital systems.

Fig.1 CFTR protein structure.^1,2

Creative Biolabs can provide a wide variety of high-quality cystic fibrosis transmembrane conductance regulator assays and products to meet the needs of drug discovery:

• Cystic Fibrosis Transmembrane Conductance Regulator Assays
• Ion Channel Cell Lines
• Ion Channel Membranes
• Membrane Protein Tools

Overview of Cystic Fibrosis Transmembrane Conductance Regulator

The cystic fibrosis transmembrane conductance regulator (CFTR) represents an ATP-binding cassette (ABC) transporter-class ion conduit responsible for the translocation of chloride ions and other ionic species across epithelial cellular membranes. Comprised of two membrane-spanning domains (MSDs), two nucleotide-binding domains (NBDs), and a singular regulatory domain (R), the CFTR protein's architecture is highly specialized. The MSDs constitute the ion channel proper, while the NBDs are tasked with ATP binding and orchestrating the channel's gating mechanisms. The regulatory domain is replete with numerous phosphorylation sites, serving as protein kinase targets for modulating CFTR functionality.

Expressed in a diverse array of tissues, including the lungs, pancreas, sweat glands, and reproductive organs, the CFTR protein is instrumental in maintaining the equilibrium of salt and water within these tissues, thereby exerting a profound influence on overall health and well-being. Genetic mutations that impede the optimal functionality of the CFTR protein disrupt this delicate balance, culminating in the accumulation of viscous, adhesive mucus within the affected tissues. Consequently, the clinical manifestations associated with cystic fibrosis arise, emphasizing the critical role of the CFTR protein in preserving homeostasis and the integrity of essential bodily systems.

Cystic Fibrosis Transmembrane Conductance Regulator Drug Discovery

In the realm of cystic fibrosis drug discovery, researchers are focusing on targeted therapies to address the underlying dysfunction of the CFTR protein. These therapeutic strategies, known as CFTR modulators, aim to restore protein function and alleviate the symptoms associated with cystic fibrosis.

Various types of CFTR modulators have been developed to target different aspects of the protein's dysfunction. These include potentiators, correctors, and amplifiers, which work synergistically to enhance the protein's activity, promote proper folding, and increase its overall expression.

By leveraging a comprehensive understanding of the CFTR protein's structure and function, scientists are making significant strides in the development of innovative treatments that target the root cause of cystic fibrosis. This promising field of research continues to evolve, offering hope for more effective and personalized therapies for those affected by this genetic disorder.

Fig.2 CFTR developmental interactome.^3,4

References

1. Harwood, Kiera H., et al. "Anti-inflammatory influences of cystic fibrosis transmembrane conductance regulator drugs on lung inflammation in cystic fibrosis." International Journal of Molecular Sciences 22.14 (2021): 7606.
2. Image retrieved from Figure 1 "“Cystic fibrosis transmembrane conductance regulator (CFTR) protein structure." Harwood, et al. 2021, used under CC BY 4.0. The original image was modified by extracting and the title was changed to " CFTR protein structure.".
3. Huang, Elena N., et al. "A developmental role of the cystic fibrosis transmembrane conductance regulator in cystic fibrosis lung disease pathogenesis." Frontiers in Cell and Developmental Biology 9 (2021): 742891.
4. Image retrieved from Figure 1 "“Cystic fibrosis transmembrane conductance regulator (CFTR) developmental interactome." Huang, et al. 2021, used under CC BY 4.0. The original image was modified by extracting and the title was changed to " CFTR developmental interactome.".