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Genetics and Evolution

Gene therapy basics Practice Questions

21 free Gene therapy basics practice questions for the USMLE Step 1, each with the correct answer and a detailed explanation. Open any question below, or take the full set as an interactive quiz.

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All Gene therapy basics questions

21 questions
  1. Q1. What is the primary goal of gene therapy?
  2. Q2. Which of the following is the most common type of delivery vector used in gene therapy?
  3. Q3. Which of the following describes an “in vivo” gene therapy approach?
  4. Q4. Which strategy describes “ex vivo” gene therapy?
  5. Q5. Which viral vector type is particularly favored for gene therapy because it typically does not integrate into the host genome, reducing risk of insertional mut…
  6. Q6. What is one major limitation of AAV vectors in gene therapy?
  7. Q7. Which of the following is a potential benefit of gene editing (e.g., using CRISPR) over traditional gene addition therapy?
  8. Q8. What is a major concern associated with genome-editing gene therapy methods like CRISPR in clinical application?
  9. Q9. Which of the following is NOT a major type of gene therapy approach?
  10. Q10. Why is tissue-specific targeting an important consideration when designing gene therapy vectors?
  11. Q11. Which of the following is a non-viral method of gene delivery in gene therapy?
  12. Q12. What is the difference between gene therapy and traditional drug therapy?
  13. Q13. Which of the following diseases would be most amenable to gene therapy using ex vivo modification of hematopoietic stem cells?
  14. Q14. What is one ethical concern unique to germline gene therapy (modifying sperm, eggs, or embryos) as opposed to somatic cell gene therapy?
  15. Q15. Which regulatory challenge is commonly associated with clinical application of gene therapy?
  16. Q16. Which of the following best describes why a “perfect vector” for gene therapy does not yet exist?
  17. Q17. How can gene therapy address diseases caused by a dominant negative mutation (where a mutant gene product interferes with normal function)?
  18. Q18. Which statement about non-viral gene delivery systems is correct compared to viral vectors?
  19. Q19. Which gene therapy approach could potentially treat a recessive loss-of-function genetic disease by restoring missing protein function?
  20. Q20. Which of the following is an advantage of using in vivo gene therapy over ex vivo gene therapy?
  21. Q21. Why is immune response a major concern in gene therapy using viral vectors?