Question 1

Which of the following best describes a therapeutic protein?

Accepted Answer

A protein engineered in the laboratory for pharmaceutical use to treat a disease or ailment.

Question 2

Which of the following is NOT an advantage of protein therapeutics compared to small molecule drugs?

Accepted Answer

High oral bioavailability

Question 3

Which of the following is a major disadvantage of protein therapeutics?

Accepted Answer

Poor membrane permeability

Question 4

Which historical event marked the first use of a recombinant protein as a therapy?

Accepted Answer

The introduction of human insulin in 1982

Question 5

Which group in the classification of protein therapies includes proteins that replace a deficient or abnormal protein?

Accepted Answer

Group I: protein therapeutics with enzymatic or regulatory activity

Question 6

Alpha-1 antitrypsin (AAT) therapy is used to treat which genetic disorder?

Accepted Answer

Alpha-1 antitrypsin deficiency (AATD)

Question 7

What is the primary function of alpha-1 antitrypsin in the lungs?

Accepted Answer

To inhibit neutrophil elastase and protect alveoli

Question 8

Which of the following best explains why recombinant Fc-AAT is considered an improvement over plasma-derived AAT?

Accepted Answer

It is more stable, more potent, and can be purified in a one-step process

Question 9

TNKase (tenecteplase) is used clinically to:

Accepted Answer

Dissolve blood clots in acute myocardial infarction

Question 10

Which structural modification in TNKase increases its resistance to PAI-1 inhibition?

Accepted Answer

Tetra alanine substitution at K296, H297, R298, and R299

Question 11

Pulmozyme (dornase alfa) is used to treat cystic fibrosis by:

Accepted Answer

Degrading extracellular DNA in mucus to reduce viscosity

Question 12

Which of the following best describes the mechanism of action of Pulmozyme in cystic fibrosis patients?

Accepted Answer

It hydrolyzes DNA in mucus, reducing its viscosity and promoting clearance

Question 13

A patient with alpha-1 antitrypsin deficiency is at increased risk for which of the following conditions?

Accepted Answer

Emphysema and liver cirrhosis

Question 14

Which of the following is a key reason why protein therapeutics are often administered by injection rather than orally?

Accepted Answer

Proteins are rapidly degraded in the gastrointestinal tract

Question 15

Which of the following best explains why monoclonal antibodies are a major class of therapeutic proteins?

Accepted Answer

They are highly specific and can target a wide range of diseases

Question 16

A new protein therapeutic is designed to deliver a cytotoxic drug specifically to cancer cells. According to the classification of protein therapies, this drug would be categorized as:

Accepted Answer

Group IIb: protein therapeutics delivering other compounds or proteins

Question 17

Which of the following best describes the role of recombinant DNA technology in protein therapeutics?

Accepted Answer

It enables the production of human proteins in non-human cells

Question 18

A patient with cystic fibrosis is prescribed Pulmozyme. Which mutation is most commonly associated with this disease?

Accepted Answer

F508del

Question 19

Which of the following equations best represents the conversion of plasminogen to plasmin in the fibrinolytic system?

Accepted Answer

$$	ext{Plasminogen} \xrightarrow{	ext{tPA}} 	ext{Plasmin}$$

Question 20

A multi-step problem: A patient with a history of smoking develops emphysema at age 40. Genetic testing reveals a mutation in the SERPINA1 gene. Which protein therapy is most appropriate, and what is the mechanism by which it treats the disease?

Accepted Answer

Alpha-1 antitrypsin (Aralast/Prolastin); it inhibits neutrophil elastase to protect alveoli

Protein Therapeutics: Biochemistry Study Guide

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