Q: Which equation best represents the probability $$P of $$successful gene delivery when the gene must overcome a mutant function and be expressed in the target cell?

$$P = p_{delivery} \times p_{expression} \times p_{function}$$

Question 1

Which of the following best describes the main goal of gene therapy in personalized medicine?

Accepted Answer

To transfer specific genes into mammalian cells to treat genetic disorders

Question 2

A patient with a genetic disorder receives gene therapy where their own cells are removed, treated in the lab, and then transplanted back. What is this approach called?

Accepted Answer

Ex vivo gene therapy

Question 3

Which of the following is a major obstacle in gene therapy related to gene expression?

Accepted Answer

Ensuring the gene is expressed and continues to be expressed

Question 4

Which viral vectors are commonly used for gene therapy?

Accepted Answer

Retroviruses and lentiviruses

Question 5

A gene therapy trial for SCID resulted in leukemia due to insertion of the therapeutic gene next to the LMO gene. What is this an example of?

Accepted Answer

Activation/mutation of an essential gene

Question 6

Which of the following is NOT a benefit of using ex vivo gene therapy?

Accepted Answer

Therapeutic DNA is distributed systemically throughout the body

Question 7

What is a GMO, and which trait is commonly engineered in crops?

Accepted Answer

A genetically modified organism; herbicide tolerance

Question 8

Which equation best represents the probability $$P of $$successful gene delivery when the gene must overcome a mutant function and be expressed in the target cell?

Accepted Answer

$$P = p_{delivery} 	imes p_{expression} 	imes p_{function}$$

Question 9

Which of the following is a real-world example of a successful gene therapy for a genetic disorder?

Accepted Answer

Restoring vision in children with Leber's congenital amaurosis

Question 10

CRISPR/Cas9 can be used in gene therapy to:

Accepted Answer

Mutate a gene to turn a gain-of-function into a loss-of-function or remove a repressor

Question 11

Which of the following is a nonviral method for gene delivery?

Accepted Answer

Fusion of cells with artificial lipid vesicles (liposomes)

Question 12

A gene therapy for sickle cell disease aims to correct a mutation in the beta-globin gene. Which of the following best describes the molecular basis of sickle cell disease?

Accepted Answer

A mutation in beta-globin causes polymerization, resulting in misshapen red blood cells

Question 13

Which of the following is a potential risk associated with gene therapy using viral vectors?

Accepted Answer

Integration into host cell's genome causing activation of oncogenes

Question 14

A gene therapy treatment for a rare form of leukemia costs $500,000. Which factor is LEAST likely to affect the price?

Accepted Answer

The color of the therapeutic vector

Question 15

Which of the following crops is an example of a GMO engineered for improved nutritional value?

Accepted Answer

Golden rice

Question 16

Which of the following is a reason why gene therapy for SCID (Severe Combined Immunodeficiency) was initially considered a breakthrough?

Accepted Answer

It was the first disease treated with gene therapy

Question 17

Which of the following best describes the difference between in vivo and ex vivo gene therapy?

Accepted Answer

In vivo introduces therapeutic DNA directly into the body; ex vivo treats cells outside the body and transplants them back

Question 18

Which of the following is a plausible ethical concern regarding the high cost of gene therapy?

Accepted Answer

It may limit access for patients who cannot afford treatment

Question 19

Which of the following is a correct application of transgenes in gene therapy?

Accepted Answer

Expressing a gene to overcome a loss-of-function mutation

Question 20

Which of the following equations best represents the cost $$C of $$gene therapy as a function of research ($$R$$), supply ($$S$$), and demand ($$D$$)?

Accepted Answer

$$C = R + S - D$$

Gene Therapy and Genetic Modification: Mechanisms, Applications, and Ethical Considerations

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