Gene therapy

Definition

Gene therapy is a medical technique that aims to treat or prevent diseases by introducing, removing, or altering genetic material within a person's cells. This approach is particularly significant for addressing single-gene disorders and can also be applied in multifactorial inheritance situations, where multiple genes and environmental factors contribute to disease development. By targeting the root cause at the genetic level, gene therapy holds the potential to correct defective genes responsible for various health issues.

5 Must Know Facts For Your Next Test

1. Gene therapy can be delivered in vivo (directly into the body) or ex vivo (cells are modified outside the body and then reintroduced).
2. One of the most common targets for gene therapy is monogenic disorders like cystic fibrosis and sickle cell disease, where a single faulty gene is responsible for the condition.
3. Gene therapy has shown promise in treating some cancers by modifying immune cells to better recognize and attack cancer cells.
4. Challenges in gene therapy include ensuring the long-term expression of the therapeutic gene, avoiding immune responses, and targeting the right cells without affecting others.
5. Regulatory agencies are closely monitoring gene therapy treatments due to ethical concerns and the potential for unintended consequences on human genetics.

Review Questions

• How does gene therapy specifically address single-gene disorders compared to multifactorial conditions?
  • Gene therapy directly targets single-gene disorders by correcting or replacing the faulty gene responsible for the disease. For example, in conditions like hemophilia, where one gene mutation leads to a lack of clotting factors, introducing a functional copy of that gene can potentially restore normal function. In contrast, multifactorial conditions involve multiple genes interacting with environmental factors, making gene therapy more complex as it may require targeting several genes simultaneously or addressing broader pathways rather than a single faulty gene.
• Discuss the role of vectors in gene therapy and how they impact the effectiveness of treatment.
  • Vectors are essential in gene therapy as they serve as delivery systems to transport therapeutic genes into target cells. Commonly used vectors include modified viruses that have been engineered to be safe for human use. The effectiveness of gene therapy largely depends on the vector's ability to efficiently deliver the gene to the right cells, express it properly, and ensure minimal immune response. The choice of vector can greatly influence treatment outcomes, making research into developing more effective and safer vectors a priority in this field.
• Evaluate the ethical considerations surrounding gene therapy and its implications for future medical practices.
  • The ethical considerations surrounding gene therapy are significant due to its potential to alter human genetics and impact future generations. Issues such as consent, equity of access to treatments, and the possibility of creating 'designer babies' raise important moral questions. Evaluating these concerns is crucial as advancements in gene therapy continue to evolve. The implications for future medical practices include establishing regulatory frameworks that balance innovation with safety and ethical responsibility, ensuring that all patients benefit equitably from these groundbreaking treatments.

Related terms

CRISPR: A revolutionary gene-editing technology that allows for precise modifications of DNA in living organisms, making it a powerful tool in gene therapy.

vector: A vehicle used to deliver therapeutic genes into target cells; common vectors include viruses that have been modified to be safe and effective for treatment.

monogenic disorders: Diseases caused by mutations in a single gene, often making them prime candidates for treatment through gene therapy.