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Allied Health Microbiology: 8.2 Gene Therapy

Allied Health Microbiology
8.2 Gene Therapy
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table of contents
  1. Cover
  2. Title Page
  3. Copyright
  4. Table Of Contents
  5. Preface
  6. Forward
  7. Chapter 1: An Invisible World
    1. 1.1 What Our Ancestors Knew
    2. 1.2 A Systematic Approach
    3. 1.3 Types of Microorganisms
    4. Summary
  8. Chapter 2: The Cell
    1. 2.1 Spontaneous Generation
    2. 2.2 Foundations of Modern Cell Theory
    3. 2.3 Unique Characteristics of Prokaryotic Cells
    4. Summary
  9. Chapter 3: Prokaryotic Diversity
    1. 3.1 Prokaryote Habitats, Relationships, and Microbiomes
    2. Summary
  10. Chapter 4: The Eukaryotes of Microbiology
    1. 4.1 Unicellular Eukaryotic Parasites
    2. 4.2 Parasitic Helminths
    3. 4.3 Fungi
    4. Summary
  11. Chapter 5: Acellular Pathogens
    1. 5.1 Viruses
    2. 5.2 The Viral Life Cycle
    3. 5.3 Prions
    4. Summary
  12. Chapter 6: Microbial Biochemistry
    1. 6.1 Microbial Biochemistry
    2. Summary
  13. Chapter 7: Microbial Growth
    1. 7.1 How Microbes Grow
    2. 7.2 Oxygen Requirements for Microbial Growth
    3. 7.3 The Effects of pH on Microbial Growth
    4. 7.4 Temperature and Microbial Growth
    5. Summary
  14. Chapter 8: Modern Applications of Microbial Genetics
    1. 8.1 Whole Genome Methods and Pharmaceutical Applications of Genetic Engineering
    2. 8.2 Gene Therapy
    3. Summary
  15. Chapter 9: Control of Microbial Growth
    1. 9.1 Controlling Microbial Growth
    2. 9.2 Testing the Effectiveness of Antiseptics and Disinfectants
    3. Summary
  16. Chapter 10: Antimicrobial Drugs
    1. 10.1 Fundamentals of Antimicrobial Chemotherapy
    2. 10.2 Mechanisms of Antibacterial Drugs
    3. 10.3 Mechanisms of Other Antimicrobial Drugs
    4. 10.4 Drug Resistance
    5. 10.5 Testing the Effectiveness of Antimicrobials
    6. 10.6 Current Strategies for Antimicrobial Discovery
    7. Summary
  17. Chapter 11: Microbial Mechanisms of Pathogenicity
    1. 11.1 Characteristics of Infectious Disease
    2. 11.2 How Pathogens Cause Disease
    3. 11.3 Virulence Factors of Bacterial and Viral Pathogens
    4. Summary
  18. Chapter 12: Disease and Epidemiology
    1. 12.1 The Language of Epidemiologists
    2. 12.2 Tracking Infectious Diseases
    3. 12.3 Modes of Disease Transmission
    4. 12.4 Global Public Health
    5. Summary
  19. Chapter 13: Innate Nonspecific Host Defenses
    1. 13.1 Physical Defenses
    2. 13.2 Chemical Defenses
    3. 13.3 Cellular Defenses
    4. 13.4 Pathogen Recognition and Phagocytosis
    5. 13.5 Inflammation and Fever
    6. Summary
  20. Chapter 14: Adaptive Specific Host Defenses
    1. 14.1 Overview of Specific Adaptive Immunity
    2. 14.2 Major Histocompatibility Complexes and Antigen-Presenting Cells
    3. 14.3 T Lymphocytes and Cellular Immunity
    4. 14.4 B Lymphocytes and Humoral Immunity
    5. 14.5 Vaccines
    6. Summary
  21. Chapter 15: Diseases of the Immune System
    1. 15.1 Hypersensitivities
    2. 15.2 Autoimmune Disorders
    3. 15.3 Organ Transplantation and Rejection
    4. Summary
  22. Chapter 16: Skin and Eye Infections
    1. 16.1 Anatomy and Normal Microbiota of the Skin and Eyes
    2. 16.2 Bacterial Infections of the Skin and Eyes
    3. 16.3 Viral Infections of the Skin and Eyes
    4. 16.4 Mycoses of the Skin
    5. 16.5 Helminthic Infections of the Skin and Eyes
    6. Summary
  23. Chapter 17: Respiratory System Infections
    1. 17.1 Anatomy and Normal Microbiota of the Respiratory Tract
    2. 17.2 Bacterial Infections of the Respiratory Tract
    3. 17.3 Viral Infections of the Respiratory Tract
    4. Summary
  24. Chapter 18: Urogenital System Infections
    1. 18.1 Anatomy and Normal Microbiota of the Urogenital Tract
    2. 18.2 Bacterial Infections of the Urinary System
    3. 18.3 Bacterial Infections of the Reproductive System
    4. 18.4 Viral Infections of the Reproductive System
    5. 18.5 Fungal Infections of the Reproductive System
    6. 18.6 Protozoan Infections of the Urogenital System
    7. Summary
  25. Chapter 19: Digestive System Infections
    1. 19.1 Anatomy and Normal Microbiota of the Digestive System
    2. 19.2 Microbial Diseases of the Mouth and Oral Cavity
    3. 19.3 Bacterial Infections of the Gastrointestinal Tract
    4. 19.4 Viral Infections of the Gastrointestinal Tract
    5. 19.5 Protozoan Infections of the Gastrointestinal Tract
    6. 19.6 Helminthic Infections of the Gastrointestinal Tract
    7. Summary
  26. Chapter 20: Circulatory and Lymphatic System Infections
    1. 20.1 Anatomy of the Circulatory and Lymphatic Systems
    2. 20.2 Bacterial Infections of the Circulatory and Lymphatic Systems
    3. 20.3 Viral Infections of the Circulatory and Lymphatic Systems
    4. 20.4 Parasitic Infections of the Circulatory and Lymphatic Systems
    5. Summary
  27. Chapter 21: Nervous System Infections
    1. 21.1 Anatomy of the Nervous System
    2. 21.2 Bacterial Diseases of the Nervous System
    3. 21.3 Acellular Diseases of the Nervous System
    4. Summary
  28. Creative Commons License
  29. Recommended Citations
  30. Versioning

8.2 Gene Therapy

Learning Objectives

  • Summarize the mechanisms, risks, and potential benefits of gene therapy
  • Identify ethical issues involving gene therapy and the regulatory agencies that provide oversight for clinical trials

Many types of genetic engineering have yielded clear benefits with few apparent risks. Few would question, for example, the value of our now abundant supply of human insulin produced by genetically engineered bacteria. However, many emerging applications of genetic engineering are much more controversial, often because their potential benefits are pitted against significant risks, real or perceived. This is certainly the case for gene therapy, a clinical application of genetic engineering that may one day provide a cure for many diseases but is still largely an experimental approach to treatment.

Mechanisms and Risks of Gene Therapy

Human diseases that result from genetic mutations are often difficult to treat with drugs or other traditional forms of therapy because the signs and symptoms of disease result from abnormalities in a patient’s genome. For example, a patient may have a genetic mutation that prevents the expression of a specific protein required for the normal function of a particular cell type. This is the case in patients with Severe Combined Immunodeficiency (SCID), a genetic disease that impairs the function of certain white blood cells essential to the immune system.

Gene therapy attempts to correct genetic abnormalities by introducing a nonmutated, functional gene into the patient’s genome. The nonmutated gene encodes a functional protein that the patient would otherwise be unable to produce. Viral vectors are sometimes used to introduce the functional gene; part of the viral genome is removed and replaced with the desired gene (Figure 8.5). More advanced forms of gene therapy attempt to correct the mutation at the original site in the genome, such as is the case with treatment of SCID.

Gene therapy using an adenovirus vector can be used to treat or cure certain genetic diseases in which a patient has a defective gene.
Figure 8.5 Gene therapy using an adenovirus vector can be used to treat or cure certain genetic diseases in which a patient has a defective gene. (credit: modification of work by National Institutes of Health)

So far, gene therapies have proven relatively ineffective, with the possible exceptions of treatments for cystic fibrosis and adenosine deaminase deficiency, a type of SCID. Other trials have shown the clear hazards of attempting genetic manipulation in complex multicellular organisms like humans. In some patients, the use of an adenovirus vector can trigger an unanticipated inflammatory response from the immune system, which may lead to organ failure. Moreover, because viruses can often target multiple cell types, the virus vector may infect cells not targeted for the therapy, damaging these other cells and possibly leading to illnesses such as cancer. Another potential risk is that the modified virus could revert to being infectious and cause disease in the patient. Lastly, there is a risk that the inserted gene could unintentionally inactivate another important gene in the patient’s genome, disrupting normal cell cycling and possibly leading to tumor formation and cancer. Because gene therapy involves so many risks, candidates for gene therapy need to be fully informed of these risks before providing informed consent to undergo the therapy.

  • Explain how gene therapy works in theory.
  • Identify some risks of gene therapy.

Annotate

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Copyright © 2019 by Open Stax and Linda Bruslind Allied Health Microbiology by Open Stax and Linda Bruslind is licensed under a Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International License, except where otherwise noted.
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