Lesson #6 - Gene Therapy and Its Potential

Gene Therapy

Introduction

  • Gene therapy involves altering genes within the body to treat disease, distinct from support groups.

  • Cancer is the second leading cause of death in the United States.

  • Traditional cancer treatments like radiation and chemotherapy have severe side effects.

  • Gene therapy offers potential as a complementary or replacement therapy for cancer.

Methods of Gene Therapy for Cancer

  • Replacement of Mutated Genetic Material:

    • Replacing mutated genetic material with healthy material.

    • Analogy: Replacing a flat tire.

  • Enhancing White Blood Cells:

    • Inserting special genes into white blood cells to detect and battle tumors.

    • Analogy: Giving white blood cells coffee and a sketch of cancer cells.

  • Reactivating Self-Destruct Mechanism:

    • Cancer cells have a deactivated self-destruct button that normally gets pressed when there's too much mutation.

    • Gene therapy can reactivate this button to eliminate cancer cells.

Delivery of Genetic Material

  • Viruses are used as double agents to deliver genetic material into cells.

    • Scientists remove the virus's DNA and insert a treatment gene.

    • The modified virus smuggles in the good genes, which are then copied during cell replication.

Example: Oligonucleotide

  • Oligonucleotide is a single-stranded piece of DNA that can be used in gene therapy.

  • It inserts itself into the mutated cell's double helix DNA, jamming up the cell's replicating gears.

  • The mutated segment of DNA cannot be transcribed or copied.

  • Potential to reverse the process, turning cancer cells back into healthy cells.

Complexity of Gene Therapy

  • Gene therapy has been used to treat simple diseases where a gene is flipped on or off.

    • Analogy: A strand of lights where one wrong bulb affects the whole string.

  • Some conditions involve complex combinations of genes being switched on or off.

  • > 20,000 proteins are involved, making it a complex problem.

Ethical and Societal Implications

  • As genetic information becomes easier to obtain, ethical questions arise.

  • Should parents be able to determine their child's eye color, height, or sexual orientation?

  • If gene therapy can cure diseases and conditions, will it drastically alter our lifespan and world population?