BIMM 110 Lecture Notes - Lecture 10: Cell Membrane, Cystic Fibrosis, Ivacaftor

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A drug (a small organic molecule) that improves the opening of the mutant cftr channel: yes, no. Introduction of a virus carrying a wild type version of cftr gene into patient"s lung cells. Ivacaftor is an example of: potentiator drug, corrector drug, gene therapy, advanced antibiotic treatment, read-through agent. Orkambi is: a corrector drug, a combination of a potentiator and a corrector drugs, advanced antibiotic treatment, used for class iii mutations, b and c. Based on this article, there are no effective gene therapies currently available for. Treating the cause: treatments that target cftr function. Gene therapy: currently mostly aims at delivering a functional cftr gene into patient"s cells (definition: introduction or alteration of genetic material into patient"s cells to cure or treat a disease) Cftr modulators: small organic drugs need to penetrate through plasma membrane, get into lung cells, bind to cftr protein change conformation correct its function. Cftr correctors: aim to increase or correct processing and delivery of.

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