Biotechnology Chapter 1 Study Guide 

Key Vocabulary

• Biotechnology: The use of living organisms or their products for human benefit.

• Fermentation: A process in which microorganisms, such as yeast, transform substances and produce useful products.

• Domestication: The process of adapting plants and animals for human use through cultivation, breeding, and selection.

• Selective breeding: Choosing organisms with desired traits to reproduce, increasing the likelihood that those traits will appear in later generations.

• Genetic engineering: The direct alteration of an organism’s genetic material to produce desired traits or products.

• Gene cloning: Making copies of a gene or DNA sequence, often to study it or use it in another organism.

• Recombinant DNA (rDNA) technology: Combining DNA from different sources to create a DNA molecule with a new sequence or function.

• Genomics: The study of an organism’s complete set of genetic material, including its genes and their interactions.

• CRISPR-Cas: A genome-editing system adapted from a natural microbial defense mechanism that can be directed to specific DNA sequences.

• Bioinformatics: The use of computing, statistics, and data analysis to organize and interpret biological information.

• Transgenic organism: An organism that contains genetic material introduced from another organism.

• Bioremediation: The use of organisms or biological processes to reduce or remove pollutants.

• Biocatalyst: An enzyme or other biological agent that speeds up a chemical reaction.

• Bioprospecting: Searching for useful biological resources or compounds, often in nature.

• Aquaculture: The cultivation of aquatic organisms, such as fish, for food or other uses.

• DNA fingerprinting: Analysis of DNA patterns to help identify an individual or determine biological relationships.

• Liquid biopsy: Analysis of biological material, such as tumor-derived DNA, in a body fluid to provide information about a disease.

• Gene-edited crop: A crop whose DNA has been altered at a targeted site using genome-editing tools; the method and resulting organism may differ from those produced by older genetic modification methods.

Key Concepts

• Biotechnology uses living organisms, cells, or products made by organisms to meet human needs. It includes both traditional practices and modern molecular techniques.

• People have used biotechnology for thousands of years. By about 2000 BC, fermentation with yeast such as Saccharomyces cerevisiae was used to make bread and alcoholic beverages. Microorganisms also contribute to foods such as cheese and yogurt. Domestication and selective breeding shaped crops and livestock.

• Corn was developed from teosinte through generations of selection and cultivation. Selective breeding also produced livestock with traits useful to people.

• Antibiotics transformed medicine in the twentieth century. Penicillin became widely used in the 1940s, when advances in large-scale batch processes helped make it available in useful quantities.

• Gene cloning, genetic engineering, and rDNA technology made it possible to produce human proteins using genetically modified organisms. These methods enabled production of medicines such as insulin, growth hormone, and clotting factors.

• The Human Genome Project, conducted from 1990 to 2003, produced a reference sequence of the human genome and advanced genomics. Genome editing has developed further through tools such as CRISPR-Cas.

• Casper, a transparent zebrafish, is an example of an organism developed to support biological research by making internal processes easier to observe.

• DIY biotechnology includes activities by biohackers and community laboratory groups. It can promote access, education, and innovation, but work outside institutional oversight may raise biosafety, biosecurity, and ethical concerns. The availability of consumer CRISPR kits, including kits marketed by the company Odin, has prompted concern about appropriate supervision and safe use.

• Biotechnology draws on multiple disciplines. In the “biotechnology tree,” basic sciences such as biology, chemistry, physics, and mathematics form the roots; genetic engineering is represented as the trunk; and diverse applications form the branches. Bioinformatics connects biological research with computing and data science.

• Genentech, founded in 1976, became a major biotechnology company. Its recombinant human insulin, approved in 1982, was the first product of modern biotechnology to reach the market.

• More than 65 percent of U.S. biotechnology companies make human medicines, and more than half of the drugs in the development pipeline target cancer. These figures illustrate the prominence of medical applications, not the full range of biotechnology.

• Biotechnology applications include:

  • Microbial biotechnology: Using microorganisms to make products, process materials, or support research.

  • Agricultural biotechnology: Developing crops with useful traits. A CRISPR-edited white button mushroom that resists browning is one example of gene editing. “Gene-edited” and “GMO” are often used differently in public discussions; the precise meaning depends on the method and context.

  • Animal biotechnology: Using genetic or reproductive technologies in animals. Examples include transgenic animals, the production of Kanuma using chicken eggs, Oxitec genetically engineered mosquitoes, efforts to develop hornless cattle, and cultured meat.

  • Forensic biotechnology: Applying DNA methods to identification and investigation, including DNA fingerprinting and some liquid-biopsy approaches.

  • Bioremediation: Using organisms to address pollution. Following the Exxon Valdez oil spill, researchers and response teams studied ways to stimulate oil-degrading bacteria such as Pseudomonas.

  • Aquatic biotechnology: Includes aquaculture and bioprospecting. AquaAdvantage salmon, a genetically engineered salmon, was approved in 2015.

  • Medical biotechnology: Developing diagnostics, medicines, and therapies.

  • Industrial biotechnology: Using biological processes in manufacturing. Enzymes in detergents, for example, act as biocatalysts that help break down stains.

• Ethical discussion should consider evidence, benefits, risks, access, environmental effects, transparency, and who is responsible for decisions. In its 2016 report, the National Academies found no substantiated evidence that foods from currently commercialized genetically engineered crops were less safe to eat than comparable non-engineered foods. This conclusion does not mean that every possible crop, use, or environmental effect has identical risks.

• Fermentation, domestication, and selective breeding: Yeast decomposes sugars for energy, releasing ethanol and carbon dioxide. Carbon dioxide makes bread dough rise; most alcohol evaporates during baking. Chinese, Greek, Roman, Babylonian, and Egyptian civilizations practiced biotechnology by about 2000 BC. Sheep and cattle domestication is a classic example; hunting and gathering is not.

  • Selective breeding examples include choosing the largest, sweetest corn ears; Figure 1.2a shows teosinte, hybrids, and modern corn (Zea mays). Turkeys have been bred for breast meat, and cows, chickens, pigs, lettuce, strawberries, cabbage, and bananas have also been shaped by breeding and selection.

  • Penicillin became widely available in the 1940s. Advances in biochemistry and cell biology in the 1950s and 1960s helped purify large amounts of antibiotics; batch processes grow cells in large quantities.

• Casper the zebrafish: Developed at Children’s Hospital Boston by mating a mutant lacking reflective pigment with one lacking black pigment, Casper helps researchers observe internal processes. It has been used in drug testing and in vivo stem-cell and cancer studies, including tracking metastasis from injected fluorescent cancer cells.

• Recombinant DNA and genomics: rDNA technology can produce insulin, human growth hormone, and blood-clotting factors, as well as disease-resistant plants, higher-yield or more nutritious crops, and pollutant-degrading bacteria. Genentech, in California, received approval in 1982 for recombinant insulin, Humulin R, widely regarded as the first modern biotechnology product. Figure 1.5 illustrates introducing a gene into cells, growing cells on plates and in a fermenter, and isolating the protein. The Human Genome Project began in 1990 and was completed in 2003; it mapped genes across 24 human chromosomes (1–22, X, and Y), and its data are freely available in public databases. Figure 1.3 shows gene maps for chromosomes 13 and 21. Synthetic genomes are another development: Johns Hopkins undergraduates built synthetic yeast chromosome 3.

• DIY biotechnology: Reports describe more than 90 percent of participants working in communal spaces, most being under 45, and about 20 percent holding doctorates. Community projects have produced inexpensive DNA-amplification instruments and malaria diagnostics, sometimes using crowdsourced funding. German authorities found antibiotic-resistant bacteria in a CRISPR kit from Odin and banned imports except to certified high-safety laboratories. With no government funding, the field is largely unregulated where activities are legal; oversight and safety remain important concerns. In 2017, a man injected himself with an unregulated HIV gene therapy, illustrating potential risks.

• The biotechnology tree and medicine: In Figure 1.4, basic sciences are the roots, genetic engineering and bioinformatics the trunk, and applications the branches: drug development, agriculture, environmental and aquatic work, detection and forensics, and biopharmaceutics and healthcare. FDA, EPA, USDA, and OSHA have regulatory roles. In 2017, 46 novel biotechnology drugs were approved, second only to 1996 with 53; oncology led with 15 (Figure 1.6). More than 350 biotechnology medicines were in development for over 200 diseases. Table 1.1 includes Humira (AbbVie; monoclonal antibody for rheumatoid arthritis and Crohn’s disease), Harvoni (Gilead; hepatitis C), Herceptin (Roche; breast cancer), Enbrel (Amgen), and Lantus (Sanofi; diabetes). Table 1.2 lists recombinant proteins including Factor VIII for hemophilia, insulin for diabetes, interferons, tissue plasminogen activator for heart attack and stroke, and monoclonal antibodies. An FDA review for 1998–2012 reported average development costs of $1.84 billion per approved biotechnology drug versus $5.67 billion per pharmaceutical drug; these estimates depend on methodology and assumptions.

• Examples across biotechnology applications:

  • Agriculture: The UN FAO estimated that feeding 9.1 billion people in 2050 would require about 70 percent more food production. Penn State researchers reported a 2016 CRISPR-edited white button mushroom (Agaricus bisporus) with an inactivated browning-enzyme gene. USDA has asked companies to advise it about gene-editing plans. Older genetic-engineering methods often inserted genes using bacteria or viruses, with less control over insertion location.

  • Animals: Transgenic goats can produce spider silk in milk. Kanuma (sebelipase alfa), made in transgenic chicken eggs, treats lysosomal acid lipase deficiency. Oxitec Friendly Aedes mosquitoes carry a larval-lethal gene and have been trialed in the Cayman Islands and Florida Keys against mosquitoes that spread dengue, Zika, chikungunya, and yellow fever. Recombinetics and UC Davis developed hornless cattle by replacing the Holstein horn gene with an Angus version; FDA regulates this application as an animal drug. Cultured beef has been reported at about $18,000 per pound, compared with $5 for lean ground beef.

  • Forensics and environmental applications: DNA fingerprinting was first used in 1987 to convict a rapist in England and is also used in paternity analysis, identifying remains, investigating wine origin and endangered-species poaching, and tracking E. coli. Liquid-biopsy companies include Illumina, Guardant Health, and Exosome Diagnostics; tests were expected to cost around $1,000. A three-gene urine test for bladder cancer was reported with 90.9 percent accuracy. After the 1989 Exxon Valdez spill in Prince William Sound, stimulating oil-degrading Pseudomonas helped shorelines clean nearly three times faster. Bioremediation was also relevant after the 2010 Deepwater Horizon spill.

  • Aquatic, medical, and industrial applications: About half of fish eaten worldwide comes from aquaculture, including trout, salmon, and catfish. AquaAdvantage salmon, approved by FDA in 2015, overproduces growth hormone and reaches market size in about half the time. Marine plankton and snails are sources of antitumor molecules. Biotechnology drugs and vaccines have helped over 325 million people. In the 1970s, enzymes began replacing phosphates in laundry detergent, reducing algal blooms.

• You Decide: genetically modified foods: Polls have found that about one-third of Americans think using science to enhance foods is wrong, with opposition reaching 50 percent in some polls. Connecticut and Maine passed labeling laws; the 2016 National Bioengineered Food Disclosure Law established federal disclosure requirements. The 2016 National Academies review examined more than 900 studies and 20 years of research and found no greater health risk for foods from the genetically engineered crops reviewed than for foods produced using other techniques. This finding does not resolve every crop-specific, environmental, labeling, or ethical question.

• Chapter Figures and Tables to Review:

  • Figures 1.1–1.6 and Tables 1.1–1.2, especially the domestication and corn examples, gene maps, biotechnology tree, recombinant-protein production, medicine examples, and drug-approval figures.

Session 1 Self-Check

Answer these prompts without looking at your notes. Then check your answers against Key Concepts and revisit any prompts you miss.

• Without looking, explain how fermentation makes bread dough rise.

• How does selective breeding differ from direct genome editing?

• Name the roots, trunk, and branches of the biotechnology tree.

• Give one example of a biotechnology application and describe its benefit.

• What is one concern to consider when evaluating a biotechnology application?

Session 2 Self-Check

Answer these prompts without looking at your notes. Then check your answers against Key Concepts and revisit any prompts you miss.

• How can genetic or biomarker information inform medical treatment, and what is one limitation?

• Explain QA versus QC in your own words.

• How does a biosimilar differ from a generic drug?

• Name two possible sources of startup funding and one risk of developing a biotechnology medicine.

• Name one biotechnology career and describe a task or skill associated with it.

Session 2 (2 hours): Medicine, Regulation, and the Biotechnology Workforce (Sections 1.2 end–1.4)

Key Vocabulary

• Stem cell: A cell that can self-renew and, depending on its type, develop into one or more specialized cell types.

• Immunotherapy: Treatment that uses or modifies components of the immune system to help fight disease.

• Gene therapy: Treatment that modifies genetic material in a patient’s cells to address a disease or its underlying cause.

• Single-nucleotide polymorphism (SNP): A common variation at a single DNA base position among individuals.

• Precision medicine: An approach to prevention or treatment that considers individual differences, including genetic, environmental, and lifestyle factors.

• Proteome: The set of proteins produced or present in a cell, tissue, or organism under particular conditions.

• Nanotechnology: The design or use of materials and devices at an extremely small scale; in medicine, it may support targeted drug delivery.

• Regenerative medicine: A field that seeks to repair, replace, or regenerate damaged cells, tissues, or organs.

• Quality assurance (QA): Systems and procedures designed to ensure that processes consistently meet established requirements.

• Quality control (QC): Testing and inspection used to determine whether materials or products meet specified standards.

• Scale-up: The process of increasing production from laboratory or pilot scale to larger manufacturing volumes.

• Contract research organization (CRO): An organization that performs research or clinical-trial services for another organization.

• Venture capital: Investment in companies with high growth potential, often in exchange for ownership shares and with substantial financial risk.

• Angel investor: An individual who invests personal funds in an early-stage company.

• Initial public offering (IPO): A company’s first offering of its shares to public investors.

• Generic drug: A drug generally developed to be equivalent to an already approved small-molecule medicine after relevant exclusivity or patent protections allow it.

• Biosimilar: A biological product shown to be highly similar to an already approved reference biologic, with no clinically meaningful differences in safety, purity, and potency.

• Organizational structure: The arrangement of teams, reporting relationships, and responsibilities within a company.

Key Concepts

• Medical biotechnology includes stem-cell research, immunotherapy, gene therapy, diagnostics, and technologies that support disease prevention and treatment.

• Personal genomics and SNP analysis can reveal genetic variation among individuals. A well-known example is the single-base change associated with sickle cell disease. A genetic result may help guide care, but it must be interpreted alongside other clinical information.

• Precision medicine aims to tailor prevention or treatment to relevant patient characteristics. Nanotechnology may assist with drug delivery, while regenerative medicine seeks to restore or replace damaged cells or tissues.

• The genome is not the whole picture: the proteome reflects proteins present at a particular time and under particular conditions. Proteomic information can help researchers investigate disease and biological responses.

• Biotechnology is heavily regulated because products and processes can affect patient safety, food and animal health, the environment, and worker safety. Depending on the product and activity, FDA, USDA, EPA, and OSHA may have roles.

  • FDA regulates many medical products, including drugs and biologics, and oversees other products within its jurisdiction.

  • USDA has responsibilities involving agriculture, plants, animals, and certain biotechnology applications.

  • EPA regulates aspects of environmental protection and certain pesticides or biotechnology-related uses.

  • OSHA addresses workplace health and safety requirements.

  • Regulatory responsibilities can overlap and depend on the product, intended use, and applicable law.

• In an arthritis scenario, a clinician might consider conventional treatments, genetic or biomarker information, and a patient’s medical history before recommending therapy. A proposed biotechnology treatment would require evidence of safety and effectiveness, and a personalized approach would not guarantee a better outcome for every patient.

• Biotechnology is both a scientific enterprise and a business. Genentech was founded in 1976 and illustrates the development of a biotechnology startup into a major company.

• Startups may seek funding from founders, angel investors, venture capital, partnerships, or public markets. Funding is used to advance research, conduct studies, establish manufacturing, and meet regulatory requirements.

• Developing a medicine can take about ten years and cost more than two billion dollars. Many startup companies do not return their investors’ capital; some estimates suggest that about 40 percent close without returns. Such figures are estimates and vary by cohort, definition, and market conditions.

• An IPO can provide a company with public investment, but it also brings disclosure requirements and public-market responsibilities.

• Biotechnology companies commonly use living organisms or biological systems to produce products, while pharmaceutical companies have traditionally emphasized chemical synthesis. The distinction is not absolute: companies may use both approaches.

• Reported estimates compare about $1.84 billion spent per approved biotechnology drug with about $5.67 billion per approved pharmaceutical drug. These averages depend on methods and assumptions; they should not be interpreted as the cost of every individual medicine.

• Generics and biosimilars are not interchangeable concepts. Generics are generally versions of chemically synthesized small-molecule drugs. Biosimilars are highly similar biological products and require comparison with a reference biologic. Competition from either can affect prices, but approval and substitution rules differ.

• A biotechnology company may include executive leadership and teams for research and development, operations, manufacturing, quality, clinical development, regulatory affairs, finance, legal work, marketing, and sales. Roles and reporting lines vary with company size and stage.

• Typical career paths include:

  • Research and development: Lab technicians, research associates, principal scientists, and senior scientists conduct experiments, analyze results, and develop products. Education may range from technical training or an associate degree to a bachelor’s, master’s, or doctoral degree, depending on the role.

  • Bioinformatics and data science: Specialists use programming, statistics, and biological knowledge to analyze genomic, clinical, or manufacturing data.

  • Operations and biomanufacturing: Teams develop processes, scale up production, operate equipment, and maintain reliable manufacturing.

  • Quality: QA develops and oversees systems and procedures; QC tests samples, materials, and products against specifications.

  • Clinical research and regulatory affairs: Clinical teams coordinate studies involving participants, while regulatory professionals prepare submissions and communicate with regulators. CROs may perform some research or trial services for biotechnology companies.

  • Business and support functions: Marketing, sales, finance, and legal teams support product strategy, operations, and compliance.

• Hiring needs change with scientific advances, funding, and product development. Employees may benefit from expertise in more than one skill area, such as biology plus data analysis or manufacturing plus quality systems. Communication, teamwork, adaptability, and careful documentation are important soft skills.

• Major U.S. biotechnology hubs include Boston, the San Francisco Bay Area, the Philadelphia–New Jersey corridor, and Research Triangle Park.

• Arthritis, genomics, and precision medicine: In the arthritis scenario, a patient’s drug is not working; genetic analysis suggests different forms of arthritis involve different genes, and a blood test might help guide drug choice. SNPs (pronounced “snips”) are single-nucleotide changes; some cause diseases such as sickle-cell anemia. Figure 1.15 illustrates three people, including one harmless SNP and one associated with a low-function protein. The proteome is the collection of proteins; the genome is not a predetermined “crystal ball.” A rough draft of the human genome was announced in February 2001. Precision medicine was formerly called pharmacogenomics.

• Medical technologies and therapies: Figure 1.16 shows nanoparticles that can deliver drugs, genes, RNA, antibodies, or immune cells to cells with target surface proteins. Gene therapy and CRISPR-Cas are sometimes described as molecular scissors; some gene therapies have cost about $1 million per treatment, and FDA has approved gene therapy for blindness. Figure 1.17 concerns stem cells. Organs on a chip and regenerative medicine are other approaches; one boy with a deadly genetic skin condition had over 80 percent of his skin restored using gene therapy combined with regenerative medicine. Many stem-cell treatments promoted for arthritis are unregulated, so patients should distinguish established evidence-based care from unapproved claims.

• Regulation and industrial biotechnology: FDA sets standards for products within its jurisdiction; USDA and EPA are often involved depending on the product and use. In 2015, FDA accepted an NDA for sensor-embedded Abilify developed by Otsuka and Proteus; the system included an ingestible sensor and a wearable patch. Industrial biotechnology has been described as having potentially greater global impact than medical or agricultural biotechnology.

• Biotechnology business and investment: Genentech was founded near San Francisco in 1976 and has been part of Roche since 2009. U.S. biotechnology revenue grew by more than 10 percent annually over a decade; worldwide biotherapeutic sales exceeded $225 billion and were reported to double about every five years. Nearly 40 percent of Americans will be diagnosed with cancer; cancer is the second leading cause of death after heart disease. About 95 percent of biotechnology companies are in North America, Europe, and Japan, across more than 54 countries; the U.S. has about 700 publicly traded biotechnology companies. Company sizes range from startups with fewer than 50 employees to large firms. At least 40 percent of startups may close without investor returns. Startups may use angel investment and venture capital; VC investment fell 46 percent from 2007 to 2008. Drug development takes about 10 years and over $2 billion. In 2013, 24 U.S. biotechnology IPOs averaged a 20 percent first-day gain and raised about $1.8 billion. Table 1.3 lists leading 2017 biotechnology companies by revenue as Amgen, Gilead, Novo Nordisk, Celgene, and Biogen, and leading pharmaceutical companies as Johnson & Johnson, Novartis, Pfizer, Roche, and AbbVie. Pharmaceutical companies such as Merck, Johnson & Johnson, and Pfizer traditionally synthesize compounds such as aspirin; biotechnology companies use living organisms, but partnerships and mixed approaches blur the distinction.

• You Decide: generics and biosimilars: Patents can last up to 20 years. Generics are cheaper versions of small-molecule drugs; biosimilars are harder-to-replicate versions of biological drugs and require costly development and comparison processes. The biosimilar market was estimated to exceed $55 billion by 2020. Global access, affordability, and pricing are important policy questions; the Martin Shkreli drug-pricing controversy is an end-of-chapter discussion example.

• Biotechnology workforce and careers: The industry employs over 200,000 U.S. workers. Biotechnology companies invest at least four times more in R&D than other high-tech industries, sometimes close to 50 percent of their budgets. A Radford/AON survey listed approximate salaries of $55,700 for Ph.D. starters, over $120,000 for senior scientists, $40,600 for M.A. roles, and $43,520 for B.A. roles; commercial pay is generally higher than academic pay. About 56 percent of students entering training programs had little science background, and the industry had tripled in size since 1992. The Orphan Drug Act is relevant to development for rare diseases. Multiple skill areas—for example, molecular biology plus an IT minor—and communication, teamwork, adaptability, and documentation skills can be valuable.

  • Research and laboratory roles: Lab technicians clean equipment and stock supplies; a B.A. or B.S. may be listed for these roles. Research associates or assistants are “bench” scientists and may have a B.S. or M.S. Principal and senior scientists, usually with Ph.D.s, lead research and may develop patents or seek grants.

  • Bioinformatics and data science: These roles combine programming, statistics, and biology. Data scientists manage large datasets and may work on cybersecurity.

  • Biomanufacturing and quality: Scaling up is like cooking for yourself versus preparing a Thanksgiving dinner for 50. Entry roles include material handler and manufacturing associate; engineers may specialize in chemical, electrical, environmental, or industrial fields. QA systems support and assure consistent final quality; QC testing checks products and materials against requirements and agency regulations. Entry roles include validation technician, documentation clerk, and QC inspector; product-complaint specialists also contribute.

  • Clinical, regulatory, and business roles: Clinical research and regulatory affairs professionals monitor compliance and trials. Sales representatives work with doctors and hospitals; marketing specialists develop campaigns; finance is often led by CFOs; legal teams handle matters such as patents.

• Biotechnology clusters: Hubs include San Francisco, San Diego, Los Angeles, Seattle, Boston/Cambridge, New York, Philadelphia (including university spin-offs), the New Jersey corridor, Maryland and northern Virginia near NIH and FDA, Raleigh-Durham Research Triangle Park (including CROs), Chicago, and Texas.

• Chapter Figures and Tables to Review:

  • Figures 1.14–1.20 and Table 1.3, especially SNPs and precision medicine, nanoparticles, stem cells and regenerative medicine, and company revenue comparisons.

Turn-In Checklist

• Vocabulary flashcards or vocabulary list.

• Types-of-biotechnology chart.

• Labeled biotechnology tree.

• Written You Decide responses.

• Answers to Study Questions 1–24.

• Career matching table.

• Two paper exit tickets.

Part 2: Study Questions

Session 1 Study Questions

1. Define biotechnology and classify bread fermentation by Saccharomyces cerevisiae as an example involving an organism, its product, or both. Explain the role of released carbon dioxide.

2. Explain how selecting teosinte plants over generations produced modern corn. How does this illustrate domestication and selective breeding rather than direct genome editing?

3. Casper zebrafish lack reflective and black pigment. Explain how their transparency can help researchers track metastasis after fluorescent cancer cells are introduced.

4. Why did penicillin’s widespread use in the 1940s depend on production advances? Describe how batch processes helped address the challenge.

5. Genentech’s recombinant Humulin R was approved in 1982. Outline the major steps shown in Figure 1.5, from introducing a gene into cells through recovering the protein, and explain why the process is recombinant DNA technology.

6. The Human Genome Project ran from 1990 to 2003. What did it contribute to genomics, and what does the reference map across 24 chromosomes (1–22, X, and Y) represent?

7. Describe one potential benefit and one biosafety or ethical concern associated with DIY biotechnology. Include the chapter’s report about an Odin CRISPR kit and antibiotic-resistant bacteria.

8. Apply the biotechnology-tree model to an example such as recombinant insulin: identify the roots, trunk, and a branch, and explain how bioinformatics connects to the model.

9. Figure 1.6 reports 46 novel biotechnology-drug approvals in 2017, including 15 oncology drugs. Calculate the oncology share as a percentage and explain what this figure does—and does not—show about biotechnology applications.

10. Use examples from Tables 1.1 and 1.2 to distinguish a named biotechnology medicine from a recombinant-protein class or use. Give two examples and identify their conditions or purposes.

11. One reported estimate puts average development cost at $1.84 billion per approved biotechnology drug versus $5.67 billion per pharmaceutical drug. Find the difference and give one reason not to treat these averages as the cost of every individual medicine.

12. Compare the CRISPR-edited non-browning mushroom with older genetic-engineering methods that inserted genes using bacteria or viruses. Why should discussions of “GMO” specify method and context?

Session 2 Study Questions

13. A single-nucleotide polymorphism (SNP) is associated with sickle cell disease. Explain what changes at the DNA level and why the example shows that a genetic variant can have a biological effect without every SNP being harmful.

14. In the arthritis scenario, a patient’s current treatment is not working. Describe how genomics or biomarker information could inform treatment choice alongside clinical evidence and patient history; identify one limitation.

15. Contrast the genome with the proteome. Why can protein measurements vary with tissue, time, or conditions even when an individual’s genome is largely unchanged?

16. Figure 1.16 shows nanoparticles used for targeted delivery. Name two kinds of cargo they may deliver and explain how target-cell surface proteins could contribute to delivery.

17. Explain how stem cells and regenerative medicine may contribute to repair or replacement of damaged tissue. Refer to the boy whose skin was restored using gene therapy combined with regenerative medicine, and explain why this case does not establish that all promoted stem-cell treatments work.

18. Why should patients be cautious about unregulated stem-cell treatments promoted for arthritis? Distinguish evidence-based care from an unapproved claim.

19. The sensor-embedded Abilify system combined an ingestible sensor and wearable patch. Explain the medical purpose of such a system and describe how FDA, USDA, EPA, and OSHA responsibilities differ in biotechnology oversight.

20. Trace possible startup funding from early-stage angel investment or venture capital to a 2013 IPO. Relate the reported 40 percent failure estimate, development timelines of about 10 years, and costs above $2 billion to investor risk.

21. Using Table 1.3 examples, compare how biotechnology and pharmaceutical companies commonly make products. Why do partnerships and mixed approaches make the distinction imperfect?

22. Distinguish a generic drug from a biosimilar. How can competition affect prices, and why are the products not simply interchangeable categories?

23. Compare the typical work and preparation of a lab technician, research associate, and principal scientist. Explain how responsibilities tend to change across these roles.

24. Use the Thanksgiving dinner analogy to explain scale-up. Then distinguish QA from QC with one manufacturing example of each.

25. The chapter reports approximate salaries of $55,700 for Ph.D. starters, over $120,000 for senior scientists, $40,600 for M.A. roles, and $43,520 for B.A. roles. Identify one cautious comparison supported by these figures and one reason they should not be treated as universal current salaries; relate your response to the chapter’s hiring trends.

26. Name two biotechnology hubs and explain how Research Triangle Park’s CROs can support biotechnology companies. What factors might influence where a company locates?