WebApr 10, 2024 · Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. WebCFTR Assays Various assays for measuring cystic fibrosis transmembrane conductance regulator (CFTR) ionchannel activity, as well as its stability in the membrane, can be used for basic research and drug discovery efforts. 5 min read In this article Western Blot Assays
Endocytic trafficking of CFTR in health and disease
WebRegulation of CFTR Trafficking by USP10. CFTR is a member of the ABC transporter superfamily and is responsible for chloride homeostasis across the cell membrane in epithelial cells. The surface expression of CFTR is tightly controlled by recycling in endosomes, where the ubiquitination of CFTR targets it to the lysosome for degradation. WebThe most common (>90%) mutation in CF, a deletion of phenylalanine 508 (ΔF508), causes improper folding of the CFTR protein, resulting in its retention in the endoplasmic reticulum and proteosomal degradation. Major CF research efforts are devoted to the attempt to rescue ΔF508-CFTR defective trafficking to restore normal epithelial function. storage units in 34986
SFPQ rescues F508del-CFTR expression and function in cystic ... - PubMed
WebApr 10, 2024 · Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein. Ivacaftor, which is known as a ... WebFeb 27, 2024 · CFTR mutations are divided into six classes determined by the specific defect in CFTR protein synthesis, trafficking, function, or stability (O’Sullivan and Freedman, 2009) (Figure 1 and Table 1), although many CFTR mutants present multiple defects, such as F508del-CFTR with deficient trafficking, function, and stability (Veit et … WebSep 2, 2011 · The most prevalent disease-causing mutation of CFTR is the deletion of Phe508 (ΔF508), which leads to defects in conventional Golgi-mediated exocytosis and cell surface expression. We report that ΔF508 … storage units in 77365