PPT-POTENTIATORS: HOW DO THEY IMPACT THE FATE OF CFTR DURING BI

1 Cellular Protein Chemistry Faculty of Science Utrecht University Padualaan 8 3584 CH Utrecht The Netherlands 2 Galapagos NV Generaal De Wittelaan L11A3 2800 Mechelen Belgium . Introduction. Conclusions. The novel . potentiator. GP-2 is highly efficacious towards enhancing CFTR function following translational RT of . PTCs, and compares favorably with . ivacaftor. , even . www.glpg.com. Defects in CFTR that result in Cystic Fibrosis can be broadly categorized into three processes: translational, folding/maturation, and functional. To. address the underlying causes affecting the folding/maturation and functional properties of CFTR, two biomolecular activities are required, namely correctors to increase CFTR levels at the cell surface, and potentiators to allow the effective opening of the CFTR channel. Combined, these activities allow chloride ion transport yielding improved hydration of the lung surface and subsequent restoration of mucociliary clearance. . CFTR-related . manifestations. Valentina . Shakhnovich. 1. , . Lakshmi . Katta. 2. , . Kathy . Christenson. 1. , . Julie . Bass. 1. , . Emily . Farrow. 1. , . Sarah . Soden. 1. , . Laurie . Smith. 3. auto-suggestion, the mystery of the past, . but mainly, fate. Let’s look at these from . least. important to . most. important.. a1. The . mystery of the past is mentioned briefly in paragraphs 10 & 13. P10 is mostly about collector and societies that focus on gun relics from the past, . with Purecol (Cedarlane, ) and cultured in complete SAGM (LHC basal medium supplemented with the SAGM kit (Clonetics, Walkersville, MD) and 25 ng/ml EGF, 100 U/ml of penicillinstreptomycin, 0.07 µg/m with Purecol (Cedarlane, ) and cultured in complete SAGM (LHC basal medium supplemented with the SAGM kit (Clonetics, Walkersville, MD) and 25 ng/ml EGF, 100 U/ml of penicillinstreptomycin, 0.07 µg/m Fate31Core31SystemA complete rewrite of Fate from the ground up Streamlines a number of prior system concepts introduces the four core actions of the game and more Turned from a single book into a rob Version 301onlineFate Core is easy to play online you dont need to rely on a Virtual TableTop VTT application at all You can rely on any video or voice chatsoftwareand augment your online experience ISI Cosupervisor Jonas Fuxe Karolinska InstitutetTitle Role of CFTR in epithelial mesenchymal transition EMT by functional genomicsObjectives To identify CFTRrelated epithelial differentiation pathway PI: John C. Kappes, PhD. Co-PI: Zhengrong Yang, PhD. Funded by the Cystic Fibrosis Foundation. C. ystic . F. ibrosis . T. ransmembrane Conductance . R. egulator (CFTR) is an ABC transporter-class ion channel protein that regulates chloride conductance across epithelial cell membranes. CRMS (CF related metabolic syndrome). CFRD (diabetes). CFRLD (Liver disease). PS (pancreatic sufficient). PI (pancreatic insufficient). 1981 average age: 18. 2016 average age: 39 (oldest 86). Not just a pediatric illness. ObjectivesUnderstand the genetic abnormality causing cystic fibrosis (CF) and the newborn screen processRecognize the multiple organ systems affected by CFIdentify common treatments /medications and m . Paediatric. Pulmonologist & Sleep physician. CF is a life-shortening, multisystem genetic disease.. First described by Dr. Dorothy Anderson (American Pathologist). provided the first description of the disorder in 1938. . DEFINITION. Most . frequent. . congenital. . metabolic. . disease. Autosomal. . recessive. inheritance – . only. a person . with. 2 . clinically. . significant. . mutations. . becomes. .