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Endocrine Pathway Models applicable for research into regulated hormone signalling systems under controlled conditions

Eckford PD, Li C, Ramjeesingh M, Bear CE (2012) Cystic fibrosis transmembrane conductance regulator (CFTR) potentiator VX-770 (ivacaftor) opens the defective channel gate of mutant CFTR in a phosphorylation-dependent but ATP-independent manner

This model was evaluated by: i) Serum ALT and bilirubin, ii) flow cytometry to define the immune cell composition in the liver and blood iii) H&E staining to assess steatosis, inflammation and ballooning iv) human/mouse aSMA and human Collagen-3 to asses mouse and human hepatic stellate cells activation and v) human CK8/18 to asses Mallory bodies formation

Furthermore, the expression of antibiotic class-specific influx channels and their functional overlap, and the fact that many metabolites impact several metabolic pathways, complicate the design of effective interventions targeting membrane permeability

Imagine the potential of certain AAV variants that can cross the blood-brain barrier, delivering therapeutic genes directly to brain cells