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💊intro to pharmacology review

Gene therapies

Written by the Fiveable Content Team • Last updated September 2025
Written by the Fiveable Content Team • Last updated September 2025

Definition

Gene therapies are medical treatments that involve altering the genes inside a patient's cells to treat or prevent disease. This innovative approach aims to correct defective genes responsible for disease development, potentially offering permanent solutions rather than temporary relief. By introducing, removing, or altering genetic material, gene therapies can address a wide range of conditions, including genetic disorders, some cancers, and viral infections.

5 Must Know Facts For Your Next Test

  1. Gene therapies can be categorized into two main types: somatic cell therapy, which targets non-reproductive cells, and germline therapy, which involves changes in reproductive cells that can be inherited.
  2. One of the most notable successes of gene therapy is the treatment of inherited retinal diseases, restoring vision in patients by targeting specific genetic defects.
  3. Challenges in gene therapy include delivery methods, immune responses, and ensuring long-term expression of the therapeutic gene within the patient's body.
  4. Regulatory approval for gene therapies has increased in recent years, with several products gaining FDA approval for treating various conditions.
  5. Ethical concerns around gene therapy include the potential for unintended consequences and the debate over germline modifications that could affect future generations.

Review Questions

  • How do gene therapies differ from traditional medical treatments in their approach to disease management?
    • Gene therapies fundamentally differ from traditional treatments as they aim to address the root cause of diseases by correcting or modifying genes rather than merely alleviating symptoms. Traditional treatments often involve medications or surgeries that provide temporary relief without changing the underlying genetic issues. In contrast, gene therapies seek to provide long-lasting or permanent solutions by fixing genetic defects directly, which could revolutionize how chronic and hereditary conditions are treated.
  • Discuss the role of vectors in the effectiveness of gene therapies and the challenges associated with their use.
    • Vectors are crucial for the success of gene therapies as they serve as delivery systems to transport therapeutic genes into target cells. Commonly used vectors include modified viruses that can efficiently penetrate cell membranes and integrate genetic material into the host's DNA. However, challenges arise due to potential immune responses against these vectors, difficulties in targeting specific cells, and ensuring the stable expression of introduced genes without causing harmful effects.
  • Evaluate the implications of recent advancements in gene therapy technologies on future treatment strategies for genetic disorders.
    • Recent advancements in gene therapy technologies, especially with tools like CRISPR, hold significant promise for revolutionizing treatment strategies for genetic disorders. These technologies allow for precise editing of genes, which not only enhances the potential effectiveness of treatments but also expands the range of conditions that can be addressed. The ability to target and modify specific genes may lead to personalized medicine approaches where therapies are tailored to individual genetic profiles, improving outcomes while minimizing side effects. However, ethical considerations surrounding these advancements necessitate careful regulation and ongoing public discourse.