Drug 2-Lumacaftor (VX-809): In people with the most common CF mutation, F508del, a series of problems prevents the CFTR protein from folding into the correct shape and reaching its proper place on the cell surface. The cell recognizes the protein as abnormal and targets it for degradation before it makes it to the cell surface. In order to treat this problem, two drugs are required - an agent to get the protein to the surface and then Ivacaftor to open the channel and increase chloride transport. The drug Lumacaftor has been identified as a treatment to help with the trafficking of the protein to the cell surface. When Lumacaftor is added to Ivacaftor, the protein gets to the surface and also increases chloride transport by increasing channel opening time. For which class(es) of mutations would Lumacaftor be most effective?
Drug 2-Lumacaftor (VX-809): In people with the most common CF mutation, F508del, a series of problems prevents the CFTR protein from folding into the correct shape and reaching its proper place on the cell surface. The cell recognizes the protein as abnormal and targets it for degradation before it makes it to the cell surface. In order to treat this problem, two drugs are required - an agent to get the protein to the surface and then Ivacaftor to open the channel and increase chloride transport. The drug Lumacaftor has been identified as a treatment to help with the trafficking of the protein to the cell surface. When Lumacaftor is added to Ivacaftor, the protein gets to the surface and also increases chloride transport by increasing channel opening time. For which class(es) of mutations would Lumacaftor be most effective?
Human Anatomy & Physiology (11th Edition)
11th Edition
ISBN:9780134580999
Author:Elaine N. Marieb, Katja N. Hoehn
Publisher:Elaine N. Marieb, Katja N. Hoehn
Chapter1: The Human Body: An Orientation
Section: Chapter Questions
Problem 1RQ: The correct sequence of levels forming the structural hierarchy is A. (a) organ, organ system,...
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Drug 2-Lumacaftor (VX-809): In people with the most common CF mutation, F508del, a series of problems prevents the CFTR protein from folding into the correct shape and reaching its proper place on the cell surface. The cell recognizes the protein as abnormal and targets it for degradation before it makes it to the cell surface. In order to treat this problem, two drugs are required - an agent to get the protein to the surface and then Ivacaftor to open the channel and increase chloride transport. The drug Lumacaftor has been identified as a treatment to help with the trafficking of the protein to the cell surface. When Lumacaftor is added to Ivacaftor, the protein gets to the surface and also increases chloride transport by increasing channel opening time.
- For which class(es) of mutations would Lumacaftor be most effective?
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