Vaccines and gene therapy represent two of the most powerful and transformative applications of biotechnology in modern medicine. While vaccines focus on preventing disease by priming the immune system, gene therapy seeks to treat or cure conditions by correcting the underlying genetic cause. For PharmD students, understanding the mechanisms, delivery systems, and clinical considerations of these advanced therapies is critical for patient education and managing the next frontier of pharmaceuticals.
1. Live-attenuated vaccines are generally contraindicated in which patient population due to the risk of causing actual disease?
- Healthy adolescents
- Patients with a fear of needles
- Immunocompromised patients
- Geriatric patients with well-controlled hypertension
Answer: Immunocompromised patients
2. Which of the following is the primary goal of gene therapy?
- To boost the immune system against a future infection
- To introduce, inactivate, or replace a gene to treat a disease
- To create a diagnostic test for a genetic disorder
- To produce large quantities of a therapeutic protein in a bioreactor
Answer: To introduce, inactivate, or replace a gene to treat a disease
3. How do mRNA vaccines, such as some COVID-19 vaccines, work?
- They introduce a weakened virus into the body
- They introduce a viral protein (antigen) to trigger an immune response
- They provide mRNA that instructs host cells to produce a specific viral antigen
- They use a harmless virus to deliver DNA into the cell nucleus
Answer: They provide mRNA that instructs host cells to produce a specific viral antigen
4. In gene therapy, what is a “vector”?
- A molecule used to carry and deliver a therapeutic gene into target cells
- The therapeutic protein product itself
- A computer program that models gene expression
- A diagnostic marker for a genetic disease
Answer: A molecule used to carry and deliver a therapeutic gene into target cells
5. A toxoid vaccine (e.g., tetanus vaccine) is made from:
- A live, weakened bacterium
- A killed, whole bacterium
- A bacterial toxin that has been inactivated
- A recombinant bacterial protein
Answer: A bacterial toxin that has been inactivated
6. What is a major advantage of using an adeno-associated virus (AAV) as a vector for gene therapy?
- It can carry very large genes
- It typically does not integrate into the host genome and has low immunogenicity
- It only infects rapidly dividing cells
- It causes a strong and desirable inflammatory response
Answer: It typically does not integrate into the host genome and has low immunogenicity
7. “Active immunity,” as conferred by a vaccine, is characterized by:
- The direct transfer of pre-made antibodies to a person
- The development of the recipient’s own immune response and memory cells
- A temporary, short-term protection against a pathogen
- The use of antitoxin to neutralize a toxin
Answer: The development of the recipient’s own immune response and memory cells
8. CAR-T cell therapy is an example of what type of gene therapy approach?
- In vivo therapy, where the vector is injected directly into the patient
- Ex vivo therapy, where patient cells are modified in the lab and then re-infused
- Germline gene therapy, which alters reproductive cells
- A non-viral delivery method using liposomes
Answer: Ex vivo therapy, where patient cells are modified in the lab and then re-infused
9. What is the function of an “adjuvant” in a vaccine?
- To preserve the vaccine during storage
- To enhance the immune response to the antigen
- To inactivate the pathogen
- To act as the primary antigen itself
Answer: To enhance the immune response to the antigen
10. A major safety concern for gene therapy vectors that integrate into the host genome, such as retroviruses, is:
- The vector being cleared too quickly from the body
- The inability to infect non-dividing cells
- Insertional mutagenesis, potentially causing cancer
- The limited size of the gene that can be carried
Answer: Insertional mutagenesis, potentially causing cancer
11. A subunit vaccine is composed of:
- The entire, killed pathogen
- A live, but weakened, version of the pathogen
- A specific, purified component of a pathogen, like a protein or polysaccharide
- An inactivated toxin
Answer: A specific, purified component of a pathogen, like a protein or polysaccharide
12. Somatic cell gene therapy aims to correct genetic defects in:
- All cells of the body, including reproductive cells
- An individual’s non-reproductive cells, and the changes are not heritable
- Only embryonic stem cells
- Germline cells (sperm or egg), making the changes heritable
Answer: An individual’s non-reproductive cells, and the changes are not heritable
13. Why are conjugate vaccines (e.g., Hib vaccine) particularly effective in young children?
- They link a polysaccharide antigen to a protein, which elicits a stronger T-cell dependent immune response
- They are administered as a nasal spray, which is easier for children
- They are made from live viruses
- They do not require any booster shots
Answer: They link a polysaccharide antigen to a protein, which elicits a stronger T-cell dependent immune response
14. Which of the following represents a significant challenge for non-viral gene delivery methods like liposomes?
- They are often highly immunogenic
- They have a high risk of integrating into the host genome
- They generally have lower efficiency of gene delivery compared to viral vectors
- They can only be used for ex vivo therapies
Answer: They generally have lower efficiency of gene delivery compared to viral vectors
15. “Herd immunity” is achieved when:
- All individuals in a population are vaccinated
- A sufficiently high proportion of a population is immune to a disease, protecting those who are not
- A disease has been completely eradicated worldwide
- A vaccine is 100% effective in every person who receives it
Answer: A sufficiently high proportion of a population is immune to a disease, protecting those who are not
16. Gene augmentation therapy is most suitable for treating diseases caused by:
- A gain-of-function mutation where a protein is overactive
- A loss-of-function mutation where a functional protein is missing or deficient
- A viral infection
- A bacterial infection
Answer: A loss-of-function mutation where a functional protein is missing or deficient
17. A key advantage of mRNA vaccine technology is:
- The extreme stability of mRNA at room temperature
- The ability to rapidly design and manufacture vaccines in response to new pathogens
- The integration of the vaccine’s genetic material into the host DNA
- The lack of a need for a delivery vehicle like a lipid nanoparticle
Answer: The ability to rapidly design and manufacture vaccines in response to new pathogens
18. What is the primary role of a pharmacist in vaccination services?
- Diagnosing infectious diseases
- Prescribing antibiotics for viral infections
- Educating patients, identifying candidates for vaccination, and administering vaccines
- Manufacturing vaccines in the community pharmacy
Answer: Educating patients, identifying candidates for vaccination, and administering vaccines
19. A key difference between a live-attenuated vaccine and an inactivated (killed) vaccine is that:
- Only the inactivated vaccine can potentially cause a mild form of the disease
- The live-attenuated vaccine can replicate in the host and typically elicits a stronger immune response
- The inactivated vaccine does not contain any antigens
- The live-attenuated vaccine does not require refrigeration
Answer: The live-attenuated vaccine can replicate in the host and typically elicits a stronger immune response
20. A significant limitation of using retroviral vectors for gene therapy is that they:
- Only infect non-dividing cells
- Cannot integrate their genetic material into the host genome
- Only infect dividing cells, limiting their use for certain tissues
- Have a very large gene carrying capacity
Answer: Only infect dividing cells, limiting their use for certain tissues
21. A patient receiving passive immunity, such as through an infusion of monoclonal antibodies, will have:
- Lifelong protection against the disease
- The ability to produce their own antibodies against the pathogen
- Immediate, but temporary, protection against a specific pathogen or toxin
- An enhanced ability to respond to future vaccines
Answer: Immediate, but temporary, protection against a specific pathogen or toxin
22. Which of the following is NOT a component of an mRNA vaccine?
- The mRNA sequence for the target antigen
- A lipid nanoparticle delivery system
- A live, attenuated virus
- Salts and sugars to maintain stability
Answer: A live, attenuated virus
23. “Gene silencing” can be achieved therapeutically using which biotechnology?
- Recombinant protein inhibitors
- Monoclonal antibodies
- RNA interference (RNAi)
- Toxoid vaccines
Answer: RNA interference (RNAi)
24. A contraindication to vaccination is a condition that:
- Greatly increases the risk of a serious adverse reaction
- Means the vaccine will be slightly less effective
- Requires the vaccine to be given in a different muscle
- Suggests the patient should wait a few days before being vaccinated
Answer: Greatly increases the risk of a serious adverse reaction
25. One of the greatest challenges facing gene therapy today is:
- A lack of potential disease targets
- The high cost of development and treatment
- The refusal of all patients to participate in clinical trials
- The inability to synthesize DNA in a lab
Answer: The high cost of development and treatment
26. The Hepatitis B vaccine is an example of what type of vaccine?
- Live-attenuated
- Inactivated whole virus
- Recombinant subunit
- Toxoid
Answer: Recombinant subunit
27. The ability of a viral vector to specifically target certain cell types is known as its:
- Tropism
- Capacity
- Pathogenicity
- Immunogenicity
Answer: Tropism
28. Why must the influenza vaccine be administered annually?
- Because vaccine-induced immunity wanes after one year
- Because the influenza virus undergoes antigenic drift, changing its surface antigens each year
- To ensure pharmaceutical companies meet their sales targets
- Because the vaccine is only sold during the fall
Answer: Because the influenza virus undergoes antigenic drift, changing its surface antigens each year
29. The first approved gene therapy in the United States, Luxturna, is used to treat a form of:
- Hemophilia
- Muscular dystrophy
- Inherited retinal disease
- Cystic fibrosis
Answer: Inherited retinal disease
30. What role does the Advisory Committee on Immunization Practices (ACIP) play?
- It manufactures all vaccines used in the United States
- It provides recommendations on the use of vaccines in the civilian population
- It funds all vaccine research and development
- It is a pharmaceutical company that sells vaccines
Answer: It provides recommendations on the use of vaccines in the civilian population
31. In ex vivo gene therapy, what is the advantage of modifying cells outside the body?
- It is much less expensive than in vivo methods
- It allows for selection and verification of correctly modified cells before returning them to the patient
- It does not require a delivery vector
- It ensures the genetic changes are passed on to the patient’s offspring
Answer: It allows for selection and verification of correctly modified cells before returning them to the patient
32. An example of passive immunity is:
- Recovery from a measles infection
- A tetanus vaccination
- An infant receiving antibodies from its mother through breast milk
- The HPV vaccine series
Answer: An infant receiving antibodies from its mother through breast milk
33. What is the primary function of the lipid nanoparticle (LNP) in an mRNA vaccine?
- To act as an adjuvant
- To protect the fragile mRNA and facilitate its entry into host cells
- To serve as the antigen
- To make the vaccine visible in the vial
Answer: To protect the fragile mRNA and facilitate its entry into host cells
34. A major hurdle for gene therapies targeting the brain is:
- The rapid division of brain cells
- The presence of the blood-brain barrier
- The lack of blood supply to the brain
- The small size of the brain
Answer: The presence of the blood-brain barrier
35. A patient with a severe allergy to eggs should be cautious with which type of vaccine, as some are produced in chicken eggs?
- mRNA vaccines
- Some formulations of the influenza vaccine
- The tetanus toxoid vaccine
- The HPV vaccine
Answer: Some formulations of the influenza vaccine
36. Zolgensma is a gene therapy that treats spinal muscular atrophy (SMA) by:
- Delivering a functional copy of the SMN1 gene using an AAV vector
- Silencing the mutated SMN1 gene
- Using CAR-T cells to attack defective motor neurons
- Administering recombinant SMN protein
Answer: Delivering a functional copy of the SMN1 gene using an AAV vector
37. Which of the following is NOT a viral vector used in gene therapy?
- Adeno-associated virus (AAV)
- Lentivirus
- Escherichia coli
- Adenovirus
Answer: Escherichia coli
38. The goal of a vaccine is to stimulate the adaptive immune system to produce:
- Pathogenic bacteria
- Memory B-cells and T-cells
- Histamine
- A fever
Answer: Memory B-cells and T-cells
39. Germline gene therapy is ethically controversial primarily because:
- It is not effective
- The genetic changes would be heritable and passed to future generations
- It can only be used to treat non-genetic diseases
- It is less precise than somatic cell therapy
Answer: The genetic changes would be heritable and passed to future generations
40. Why would a “booster” dose of a vaccine be required?
- To treat an active infection
- To increase an immune response that has waned over time
- Because the first dose was administered incorrectly
- To protect against a completely different virus
Answer: To increase an immune response that has waned over time
41. Liposomes are used as non-viral vectors in gene therapy because their lipid bilayer can:
- Integrate into the host DNA
- Fuse with the cell membrane to deliver their contents into the cytoplasm
- Replicate independently within the cell
- Cut the target gene at a specific location
Answer: Fuse with the cell membrane to deliver their contents into the cytoplasm
42. Which vaccine type is generally considered the safest for an immunocompromised individual?
- Live-attenuated
- Recombinant subunit
- Viral vector
- It is not safe for them to receive any vaccines
Answer: Recombinant subunit
43. The “carrying capacity” of a gene therapy vector refers to:
- The maximum size of the therapeutic gene it can hold
- The number of patients it can treat
- The electrical charge of the vector
- The number of cells it can infect
Answer: The maximum size of the therapeutic gene it can hold
44. Which statement accurately describes the Vaccine Adverse Event Reporting System (VAERS)?
- It is a system that proves a vaccine caused a specific adverse event
- It is a national early warning system to detect possible safety problems with U.S.-licensed vaccines
- It is used to schedule vaccination appointments
- It is managed by pharmaceutical companies to track their sales
Answer: It is a national early warning system to detect possible safety problems with U.S.-licensed vaccines
45. A potential advantage of using lentiviral vectors over retroviral vectors is that lentiviruses can:
- Infect both dividing and non-dividing cells
- Carry much smaller genes
- Avoid integration into the host genome
- Be administered orally
Answer: Infect both dividing and non-dividing cells
46. The Human Papillomavirus (HPV) vaccine is a prophylactic vaccine designed to:
- Treat active cervical cancer
- Prevent infection with HPV types that are a major cause of cervical cancer
- Boost the immune system against the common cold
- Be administered only to males
Answer: Prevent infection with HPV types that are a major cause of cervical cancer
47. The success of a gene therapy treatment is highly dependent on:
- The patient’s blood type
- The efficient and targeted delivery of the therapeutic gene to the correct cells
- The color of the viral vector solution
- The time of day the therapy is administered
Answer: The efficient and targeted delivery of the therapeutic gene to the correct cells
48. An inactivated polio vaccine (IPV) contains:
- A live, weakened poliovirus
- Poliovirus that has been killed and cannot replicate
- A recombinant poliovirus protein
- An inactivated polio toxin
Answer: Poliovirus that has been killed and cannot replicate
49. What is a primary challenge for therapies using RNA interference (RNAi)?
- The RNA molecules are too stable in the body
- Ensuring the specific delivery of the small RNA molecules to the target tissue
- The effect is permanent and cannot be reversed
- It only works for bacterial infections
Answer: Ensuring the specific delivery of the small RNA molecules to the target tissue
50. The convergence of vaccine and gene therapy technologies is best exemplified by:
- Toxoid vaccines
- Viral vector and nucleic acid (mRNA/DNA) vaccines
- Whole-cell inactivated vaccines
- Live-attenuated vaccines
Answer: Viral vector and nucleic acid (mRNA/DNA) vaccines
I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.
Mail- Sachin@pharmacyfreak.com