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A new drug therapy regime for the treatment of cystic fibrosis employs a cocktail of three small molecules encased in a liposome that can be targeted to patients' lung cells

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A new drug therapy regime for the treatment of cystic fibrosis employs a cocktail of three small molecules encased in a liposome that can be targeted to patients' lung cells. The liposome can be taken up by the affected lung cells by endocytosis. The small molecules can then act like mini-mechanics: they act to repair the misfolded CFTR protein, thereby restoring the balance of chloride ions and reducing harmful mucus build-up. To learn more about this treatment, watch the YouTube video "How ORKAMBI (lumacaftor/ivacaftor) Works" using this link: https://www.orkambi.com/how-orkambi-works. The important parts of the video are 1:27 -4:58. Then answer the questions below. 6a. Why does the F508-del mutation cause "deformed" CFTR proteins that do not reach the plasma membrane? [2 pts] 6b. The new drug therapy regime includes ORKAMBI and a secret third drug. It is not yet known what the mechanism of action of this mysterious third drug is. Propose one specific mechanism that the third drug could correct in F508-del cystic fibrosis patients. [3 pts] 6c. The CFTR, which is dysfunctional in cystic fibrosis patients, is a plasma membrane protein. However, cancer can be caused by a mutation in the gene for p53, which is a transcription factor found in the cell nucleus. Some cancers form because the p53 protein that is produced is "deformed" and therefore non- functional. Could liposomes capable of entering the endomembrane system that contain mini- mechanics be used to repair the defective p53 protein and destroy the cancer cells? Why or why not?