1. What is gene therapy?
A) A method to edit RNA molecules B) A technique to replace defective genes C) A way to amplify DNA sequences D) A diagnostic tool for genetic disorders
Answer: B) A technique to replace defective genes
2. Which vector is commonly used in gene therapy?
A) Bacteria B) Viruses C) Plasmids D) Ribosomes
Answer: B) Viruses
3. What is the goal of somatic gene therapy?
A) To modify gametes B) To edit DNA in embryos C) To treat existing genetic conditions D) To introduce foreign genes for enhancement
Answer: C) To treat existing genetic conditions
4. Which disease is primarily targeted by gene therapy?
A) Diabetes B) Cystic fibrosis C) Malaria D) Osteoarthritis
Answer: B) Cystic fibrosis
5. In vivo gene therapy involves?
A) Modifying genes outside the body B) Direct delivery of genes into the body C) Amplifying genes in a laboratory D) Editing genes at the zygote stage
Answer: B) Direct delivery of genes into the body
6. What is an example of ex vivo gene therapy?
A) Inserting genes into bone marrow cells in the lab B) Injecting therapeutic genes into the bloodstream C) Targeting muscle cells with DNA vaccines D) Editing genes in embryonic cells
Answer: A) Inserting genes into bone marrow cells in the lab
7. What type of virus is often used in gene therapy?
A) Influenza virus B) Retrovirus C) Rabies virus D) Adenovirus
Answer: B) Retrovirus
8. What is a significant ethical concern in germline gene therapy?
A) Limited efficacy B) Safety in somatic cells C) Altering heritable traits D) High cost of treatment
Answer: C) Altering heritable traits
9. CRISPR-Cas9 is used in gene therapy for?
A) Protein synthesis B) Cutting specific DNA sequences C) Blocking RNA translation D) Inactivating viruses
Answer: B) Cutting specific DNA sequences
10. What is the role of liposomes in gene therapy?
A) As vectors for DNA delivery B) To modify RNA molecules C) To bind to proteins in the cell D) As a diagnostic marker for disease
Answer: A) As vectors for DNA delivery
11. What does the term “off-target effects” refer to?
A) Successful DNA editing B) Unintended gene modifications C) Efficient vector delivery D) Enhanced immune response
Answer: B) Unintended gene modifications
12. Which of the following is NOT a limitation of gene therapy?
A) High cost B) Risk of immune response C) Lack of target specificity D) Amplification of DNA sequences
Answer: D) Amplification of DNA sequences
13. Which cells are targeted in somatic gene therapy?
A) Germ cells B) Embryonic cells C) Somatic cells D) Zygote cells
Answer: C) Somatic cells
14. What is the major advantage of non-viral vectors?
A) High efficiency B) Reduced immune response C) Long-term gene expression D) High cost
Answer: B) Reduced immune response
15. What is the purpose of gene silencing?
A) To enhance gene expression B) To block protein synthesis C) To destroy defective genes D) To amplify DNA sequences
Answer: B) To block protein synthesis
16. Which technique is used to assess the success of gene therapy?
A) PCR B) ELISA C) Western blotting D) Chromatography
Answer: A) PCR
17. What is the major challenge in delivering therapeutic genes?
A) Finding target cells B) High replication of vectors C) Precise targeting and stability D) Low availability of therapeutic genes
Answer: C) Precise targeting and stability
18. What does “somatic gene therapy” focus on?
A) Preventing genetic diseases in offspring B) Correcting genes in somatic cells C) Enhancing hereditary traits D) Modifying germline cells
Answer: B) Correcting genes in somatic cells
19. Which condition was treated in the first human gene therapy trial?
A) Hemophilia B) Severe Combined Immunodeficiency (SCID) C) Huntington’s disease D) Parkinson’s disease
Answer: B) Severe Combined Immunodeficiency (SCID)
20. How does a lentivirus vector work in gene therapy?
A) By integrating into the host genome B) By directly modifying RNA C) By producing proteins D) By silencing other vectors
Answer: A) By integrating into the host genome
21. Ethical concerns in gene therapy include?
A) Cost of equipment B) Accessibility to all patients C) Altering human identity D) Both B and C
Answer: D) Both B and C
22. Which enzyme is essential for retroviral vectors?
A) DNA polymerase B) Reverse transcriptase C) RNA polymerase D) Ligase
Answer: B) Reverse transcriptase
23. What is the “knock-in” approach in gene therapy?
A) Removing faulty genes B) Introducing functional genes C) Silencing target genes D) Blocking protein production
Answer: B) Introducing functional genes
24. A major limitation of germline therapy is?
A) Temporary effects B) Ethical and safety concerns C) High specificity D) No risk to offspring
Answer: B) Ethical and safety concerns
25. What is the function of TALENs in gene therapy?
A) Gene silencing B) DNA cleavage at specific sites C) RNA degradation D) Protein modification
Answer: B) DNA cleavage at specific sites
26. How does gene therapy help in cancer treatment?
A) By killing cancer cells directly B) By correcting genetic mutations C) By silencing oncogenes D) Both B and C
Answer: D) Both B and C
27. Which factor increases efficiency in gene delivery?
A) Random integration B) Use of target-specific vectors C) High vector dose D) Using non-specific promoters
Answer: B) Use of target-specific vectors
28. The term “transfection” refers to?
A) Gene transfer in non-viral vectors B) Viral infection of cells C) Immune response to vectors D) Protein expression modification
Answer: A) Gene transfer in non-viral vectors
29. What is “zinc-finger nucleases (ZFNs)” used for?
A) RNA sequencing B) Specific DNA editing C) Enhancing protein production D) Blocking immune responses
Answer: B) Specific DNA editing
30. What is the role of bioethics in gene therapy?
A) Enhancing technological innovations B) Guiding responsible application of therapies C) Limiting medical research D) Promoting unrestricted use of gene editing
Answer: B) Guiding responsible application of therapies
