Gene pharming, a cutting-edge field within biotechnology, utilizes transgenic organisms to produce valuable pharmaceuticals. This method of creating recombinant proteins in hosts like goats or plants presents a fascinating alternative to conventional bioreactor-based systems. For PharmD students, understanding the principles, advantages, and challenges of gene pharming is key to appreciating the complex origins and unique properties of some of the biotherapeutics that are shaping the future of medicine.
1. Gene pharming is best defined as the use of which of the following to produce pharmaceuticals?
- Traditional chemical synthesis
- Transgenic animals and plants
- Large-scale bacterial fermentation only
- Human blood donors
Answer: Transgenic animals and plants
2. Which biological fluid is most commonly used as a “bioreactor” for producing recombinant proteins in transgenic animals like goats and sheep?
- Blood
- Urine
- Saliva
- Milk
Answer: Milk
3. What is a primary advantage of using transgenic plants for protein production compared to animal systems?
- Faster development time from gene to first harvest
- Lower cost for large-scale production and easier scale-up
- Identical post-translational modifications to humans
- Lower risk of environmental contamination
Answer: Lower cost for large-scale production and easier scale-up
4. The first drug produced in a transgenic animal to be approved by the FDA was ATryn®. What protein is it?
- Insulin
- Human growth hormone
- Antithrombin III
- Erythropoietin
Answer: Antithrombin III
5. A major ethical concern specifically related to gene pharming in animals is:
- The potential for gene flow into wild populations
- The cost of the final drug product
- The welfare of the transgenic animals
- The risk of bacterial contamination
Answer: The welfare of the transgenic animals
6. To ensure a recombinant protein is expressed only in the mammary gland of a transgenic animal, the gene of interest is typically linked to a:
- Ubiquitous promoter
- Mammary-specific promoter (e.g., from a milk protein gene like casein)
- Viral promoter
- Bacterial promoter
Answer: Mammary-specific promoter (e.g., from a milk protein gene like casein)
7. Which method is commonly used to create a transgenic animal?
- Exposing an adult animal to radiation
- Injecting the desired gene into the muscle tissue of an animal
- Microinjecting a DNA construct into a fertilized egg (zygote)
- Feeding the animal a diet rich in foreign DNA
Answer: Microinjecting a DNA construct into a fertilized egg (zygote)
8. A significant challenge in purifying a recombinant protein from milk is:
- The protein is usually denatured
- The milk is a very simple, pure solution
- The high concentration of other proteins and fats in milk
- The milk is produced in very small volumes
Answer: The high concentration of other proteins and fats in milk
9. What is a primary containment concern associated with using transgenic plants like corn to produce pharmaceuticals?
- The plants may escape and grow in the wild
- Cross-pollination with conventional corn crops intended for the food supply
- The plants may attract different insects
- The pharmaceuticals might be toxic to the plant itself
Answer: Cross-pollination with conventional corn crops intended for the food supply
10. What is a key advantage of using transgenic animals for producing complex proteins over bacterial systems like E. coli?
- Animals grow faster than bacteria
- Animals can perform complex post-translational modifications, such as glycosylation
- The purification process from milk is much simpler
- The cost of creating a transgenic animal is lower than culturing bacteria
Answer: Animals can perform complex post-translational modifications, such as glycosylation
11. The term “transgene” refers to:
- A gene that is naturally found in the host organism
- The foreign or modified gene that is inserted into an organism
- A gene that regulates the cell cycle
- A gene found only in viruses
Answer: The foreign or modified gene that is inserted into an organism
12. Why are chickens considered a promising bioreactor for gene pharming?
- They have a very long reproductive cycle
- Recombinant proteins can be expressed in their eggs
- They produce milk in large quantities
- Their eggs contain no other proteins
Answer: Recombinant proteins can be expressed in their eggs
13. Agrobacterium tumefaciens is a bacterium often used as a vector for what purpose?
- To produce proteins in large-scale fermenters
- To transfer genes into plants
- To act as a probiotic in transgenic animals
- To purify proteins from milk
Answer: To transfer genes into plants
14. A potential disadvantage of plant-based systems compared to mammalian systems is that:
- Plants cannot produce any proteins
- Plant glycosylation patterns can be different from human patterns, potentially causing immunogenicity
- Plants cannot be grown in large quantities
- It is impossible to extract proteins from plant tissues
Answer: Plant glycosylation patterns can be different from human patterns, potentially causing immunogenicity
15. What does the term “molecular farming” typically refer to?
- The use of molecular tools to improve traditional agriculture
- The production of pharmaceuticals in transgenic plants
- The use of bacteria to produce industrial chemicals
- A computer simulation of farming practices
Answer: The production of pharmaceuticals in transgenic plants
16. Compared to building a large-scale mammalian cell culture facility, a major economic advantage of gene pharming in animals is:
- The initial development of the transgenic line is very fast
- Scale-up can be achieved by simply breeding more animals
- No purification of the final product is required
- The animals require no special food or housing
Answer: Scale-up can be achieved by simply breeding more animals
17. The first step in creating a transgenic animal for gene pharming is to:
- Identify and isolate the gene that codes for the desired therapeutic protein
- Select the animal for breeding
- Build a specialized barn for housing
- Design the purification protocol
Answer: Identify and isolate the gene that codes for the desired therapeutic protein
18. A “biolistic” or “gene gun” approach is a method used to:
- Purify proteins from a complex mixture
- Create transgenic animals
- Physically shoot DNA-coated particles into plant cells
- Sequence a plant’s genome
Answer: Physically shoot DNA-coated particles into plant cells
19. Which of the following is NOT a potential product of gene pharming?
- Monoclonal antibodies
- Hormones
- Vaccines
- A small-molecule drug like aspirin
Answer: A small-molecule drug like aspirin
20. A significant disadvantage of using transgenic animals for drug production is the:
- Short lifespan of the animals
- Inability to produce complex proteins
- Long time required for breeding and herd scale-up
- Very low yield of protein per animal
Answer: Long time required for breeding and herd scale-up
21. Expression of therapeutic proteins in plant seeds offers what advantage?
- The protein is easy to see and extract
- The proteins are often more stable during long-term storage within the seed
- Seeds contain no native proteins or oils
- The plants can be grown in any climate
Answer: The proteins are often more stable during long-term storage within the seed
22. To confirm that a transgene has been successfully integrated into an animal’s genome, which technique would be used?
- ELISA
- Western Blot
- PCR or Southern Blot
- Chromatography
Answer: PCR or Southern Blot
23. Why is tobacco a frequently used plant for molecular pharming research?
- It is a major food crop
- It is easy to genetically transform and produces large amounts of biomass quickly
- It has a very slow growth cycle
- Its genome is not well understood
Answer: It is easy to genetically transform and produces large amounts of biomass quickly
24. The regulatory pathway for a drug produced via gene pharming is overseen by:
- The Department of Agriculture exclusively
- The Environmental Protection Agency exclusively
- The Food and Drug Administration (FDA)
- Local farm bureaus
Answer: The Food and Drug Administration (FDA)
25. A founder animal in a transgenic herd is:
- The oldest animal in the herd
- The first-generation animal that successfully incorporates and expresses the transgene
- An animal that has rejected the transgene
- The animal used for companionship
Answer: The first-generation animal that successfully incorporates and expresses the transgene
26. One strategy to prevent the spread of transgenes from pharmaceutical crops is to:
- Use plants that are male-sterile or do not produce pollen
- Plant them next to food crops to encourage cross-pollination
- Harvest the plants before they flower
- Water the plants with a special sterilizing solution
Answer: Use plants that are male-sterile or do not produce pollen
27. What is a key reason that expressing proteins in milk is preferred over expressing them in the blood of an animal?
- Blood is more difficult to collect than milk
- Milk is a less complex fluid than blood
- Proteins in the blood could have unintended physiological effects on the host animal
- Proteins are less stable in blood
Answer: Proteins in the blood could have unintended physiological effects on the host animal
28. Which of the following is an example of a potential plant-made pharmaceutical?
- Edible vaccines
- Plastic polymers
- High-fructose corn syrup
- Synthetic pesticides
Answer: Edible vaccines
29. A significant hurdle for the widespread adoption of gene pharming has been:
- A lack of interest from pharmaceutical companies
- The complete absence of any successful products
- Public perception and complex regulatory hurdles
- The technology being too simple to patent
Answer: Public perception and complex regulatory hurdles
30. The process of removing the desired protein from all other components of milk or plant tissue is known as:
- Transformation
- Fermentation
- Purification
- Transcription
Answer: Purification
31. The primary purpose of creating a transgenic mouse in a research setting is often to:
- Produce large quantities of a therapeutic drug
- Serve as a disease model to study human illness
- Create a new type of pet
- Test new types of animal feed
Answer: Serve as a disease model to study human illness
32. Which of the following is a limitation of gene pharming?
- It is not possible to create monoclonal antibodies this way
- The protein yields are consistently too low to be commercially viable
- The final product is a complex mixture that requires extensive purification
- The technology is not based on recombinant DNA principles
Answer: The final product is a complex mixture that requires extensive purification
33. The use of safflower to produce recombinant human insulin demonstrates the potential of:
- Animal-based bioreactors
- Plant-based molecular farming
- Bacterial fermentation
- Marine biotechnology
Answer: Plant-based molecular farming
34. When compared to CHO cell culture, the capital cost of establishing a large herd of transgenic goats is generally:
- Significantly higher
- Roughly the same
- Significantly lower
- Not a relevant economic factor
Answer: Significantly lower
35. A “knock-in” animal is one where:
- A gene has been deleted from the genome
- A gene has been inserted at a specific location in the genome
- The animal’s immune system has been disabled
- The animal has been cloned
Answer: A gene has been inserted at a specific location in the genome
36. A key feature of a therapeutic protein that dictates the need for a eukaryotic expression system (like an animal or plant) is the:
- Small size of the protein
- Simple, linear structure
- Requirement for complex folding and post-translational modifications
- High solubility in water
Answer: Requirement for complex folding and post-translational modifications
37. Before a drug from a transgenic source can be approved, manufacturers must prove:
- That the transgenic animal is a new species
- That the protein product is consistent across different batches and different animals/generations
- That the drug can be produced faster than any other method
- That the public fully supports the technology
Answer: That the protein product is consistent across different batches and different animals/generations
38. Which of these is a major difference between a drug from a transgenic animal and a biosimilar of that drug?
- The biosimilar would be produced in a different system (e.g., CHO cells)
- The biosimilar is a small molecule
- The original drug does not require purification
- The biosimilar is not a protein
Answer: The biosimilar would be produced in a different system (e.g., CHO cells)
39. One reason for the limited number of approved “pharmed” products is:
- The technology does not work
- The high efficiency and low cost of existing mammalian cell culture systems for many products
- A complete lack of regulatory guidelines
- The proteins produced are always toxic
Answer: The high efficiency and low cost of existing mammalian cell culture systems for many products
40. If a therapeutic protein was successfully produced in rice seeds, what would be a major advantage for global health applications?
- The rice could be cooked and eaten as an edible vaccine
- The stability of the protein in the seed could reduce the need for a cold chain
- The rice would be resistant to all crop diseases
- The protein would be easy to extract in a home kitchen
Answer: The stability of the protein in the seed could reduce the need for a cold chain
41. The DNA construct used to create a transgenic animal must contain the gene of interest and what other critical element?
- A promoter sequence to drive expression in the target tissue
- An antibiotic resistance gene
- A gene for a fluorescent protein
- A sequence that makes the DNA circular
Answer: A promoter sequence to drive expression in the target tissue
42. Which class of biopharmaceuticals is a major target for production via gene pharming due to their complexity and high demand?
- Peptide hormones
- Monoclonal antibodies
- Small interfering RNAs (siRNAs)
- Synthetic vitamins
Answer: Monoclonal antibodies
43. A major downstream processing step for a protein from a plant source would be the removal of:
- Animal viruses
- Plant-specific alkaloids and phenols
- Milk fats and casein
- Bacterial endotoxins
Answer: Plant-specific alkaloids and phenols
44. The consistency of protein production from a transgenic animal can be affected by the animal’s:
- Diet, health, and lactation cycle
- Favorite color
- Time spent sleeping
- Name given by the farmer
Answer: Diet, health, and lactation cycle
45. One of the earliest examples of a protein produced in the milk of transgenic animals for therapeutic use was:
- Alpha-1-antitrypsin for treating emphysema
- Human hemoglobin as a blood substitute
- Myostatin inhibitor for muscle growth
- Spider silk for industrial use
Answer: Alpha-1-antitrypsin for treating emphysema
46. The main reason gene pharming is not used for small-molecule drugs is that:
- Small molecules are proteins
- Small molecules are more efficiently and cheaply made by chemical synthesis
- Transgenic animals cannot produce small molecules
- The FDA prohibits it
Answer: Small molecules are more efficiently and cheaply made by chemical synthesis
47. From a pharmacist’s perspective, the origin of a recombinant protein (e.g., CHO cells vs. goat milk) is important because it can influence:
- The drug’s color and taste
- The final product’s glycosylation pattern and potential immunogenicity
- The expiration date exclusively
- The shape of the tablet or vial
Answer: The final product’s glycosylation pattern and potential immunogenicity
48. Why is it important to use a promoter specific to mammary tissue when producing a protein in milk?
- To prevent the protein from being expressed in other tissues where it could be harmful
- To make the animal produce more milk
- To ensure the protein is blue
- It is not important; any promoter will work
Answer: To prevent the protein from being expressed in other tissues where it could be harmful
49. If a transgenic plant producing a vaccine was accidentally mixed with a food crop, the primary risk would be:
- Unintended oral immunization or allergic reactions in the public
- The food crop becoming more nutritious
- The vaccine losing its potency
- The food crop tasting better
Answer: Unintended oral immunization or allergic reactions in the public
50. Overall, gene pharming represents a technology platform that:
- Has replaced all other methods of drug production
- Is best suited for all types of pharmaceutical products
- Offers a specialized, alternative production system with unique benefits and challenges
- Has been proven to be a failure with no future potential
Answer: Offers a specialized, alternative production system with unique benefits and challenges
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