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Working this is just an image of the ABC transporter and as the name intends it transports things from the inside of the cell to the outside of the cell one of those molecules that it transports is cyclic AMP it can do some other things but I'm only going to focus on cyclic AMP within the plasma membrane of an epithelial cell in close proximity is CFTR and this is that gene that is there are variants of that can lead to cystic fibrosis and CFTR is also a transporter it's actually an ABC transporter as well and it transports chloride out of the cell now in in the individuals that do not have cystic fibrosis chloride will go out of the cell and then that will lead to water going out of the cell and that will lead to hydration of the airway surface lining fluid and non neck normal mucus whereas if this isn't working properly then what happens is the mucus chloride doesn't go out water doesn't go out and the mucus becomes dehydrated and sticky I've drawn a red peon here because this is a phosphorylation site which influences the activity of this transporter.





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Genes there that's one thing but it's not giving me function I need to see that it's either working normally or so I say that these are both ABC transporters so they're linked actually at genetically because they they are derived they believe to be derived from each other over evolution but they're also linked within the cell membrane physically and here's a scaffolding protein that links these two proteins together ABC c4 and CFTR and what I've said to you without showing the data is that ABC C 4 transports cyclic AMP outside of the cell and I'm going to show you why that's important in the context of cystic fibrosis so this is just some data where I've been working on developing some novel ABC c4 inhibitors so I'm just calling it a b c c4 inhibitor because of confidentiality reasons that I'm not showing you any structure but what we're able to do is we have an assay that measures the amount of cyclic AMP that goes outside of an epit
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