Imagine that you belong to a group of scientists in a biotech company who are evaluating the potential of several therapies for the most common…

A. small chemical, administered as a pill that is a strong inhibitor of protein degradation.

Your evaluation:

Does the therapy treat the underlying molecular defect?

Is the method of delivery of the therapy reasonable: is the therapy likely to reach the affected tissues?

Is the therapy likely to have significant side effects? Explain.

Suggest at least one improvement to this therapy.

An antibody, administered as a pill that binds to the extracellular part of CFTR and changes its conformation, making it more likely to be open.

Your evaluation:

Does the therapy treat the underlying molecular defect?

Is the method of delivery of the therapy reasonable: is the therapy likely to reach the affected tissues?

Is the therapy likely to have significant side effects? Explain.

Suggest at two improvements to this therapy.

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