A pharmaceutical researcher develops a therapy to treat a protein misfolding disorder. Specifically, a mutation in this disorder leads to abnormal protein folding and subsequent intracellular degradation of the protein before it can reach the cell membrane. With the new combination drug therapy, the first drug corrects the processing and trafficking of the protein, enabling it to reach the cell surface membrane. Once the protein has reached the cell surface, its function is enhanced by the second drug. This therapy is most likely to be helpful in which of the following conditions?

Question

A pharmaceutical researcher develops a therapy to treat a protein misfolding disorder.  Specifically, a mutation in this disorder leads to abnormal protein folding and subsequent intracellular degradation of the protein before it can reach the cell membrane.  With the new combination drug therapy, the first drug corrects the processing and trafficking of the protein, enabling it to reach the cell surface membrane.  Once the protein has reached the cell surface, its function is enhanced by the second drug.  This therapy is most likely to be helpful in which of the following conditions?

Quizplus · Accepted Answer

Eductional Objective: F508 is the most common mutation in the cystic fibrosis transmembrane regulator (CFTR) protein in patients with cystic fibrosis.  This mutation leads to protein misfolding and failure of glycosylation, followed by proteasome-mediated degradation and significantly decreased number of transmembrane CFTR proteins.

A Pharmaceutical Researcher Develops a Therapy to Treat a Protein