Gene therapy

Definition

Gene therapy is a medical technique that aims to treat or prevent disease by modifying the genes within an individual's cells. This process can involve inserting, altering, or removing genes to correct genetic disorders or enhance the body's ability to fight diseases, such as cancer. Gene therapy is closely related to advancements in genetic engineering and raises significant bioethical considerations regarding its use and potential implications for society.

5 Must Know Facts For Your Next Test

1. Gene therapy has the potential to treat a wide range of conditions, including genetic disorders, some types of cancer, and viral infections.
2. The first successful gene therapy procedure took place in 1990, when a girl with a rare genetic disorder was treated by correcting a defect in her immune system.
3. There are currently several gene therapy products approved for use in various countries, showing promising results in clinical trials.
4. Gene therapy raises ethical concerns regarding safety, informed consent, and the potential for 'designer babies' if applied to germline cells.
5. Ongoing research focuses on improving delivery methods for gene therapy, such as using viral vectors or nanoparticles to transport therapeutic genes into target cells.

Review Questions

• What are the key differences between somatic and germline gene therapy, and what implications do these differences have for patients?
  • Somatic gene therapy targets non-reproductive cells, meaning any changes made will affect only the individual receiving treatment and cannot be inherited. In contrast, germline gene therapy alters reproductive cells, which means the modifications could be passed on to future generations. The implications of these differences are significant; somatic therapy might be more ethically acceptable as it doesn't affect the genetic makeup of future offspring, while germline therapy raises concerns about long-term effects on humanity and the potential for unintended consequences.
• Discuss the ethical considerations surrounding gene therapy, particularly in relation to potential risks and societal impact.
  • The ethical considerations surrounding gene therapy are multifaceted and include issues of safety, informed consent, and accessibility. Risks associated with gene therapy can include unintended genetic changes or immune reactions. Additionally, societal implications arise from the potential for gene editing technologies to widen disparities in healthcare access and create ethical dilemmas around 'designer babies,' where individuals could select traits for their children. These discussions emphasize the need for robust regulatory frameworks and public discourse on the implications of such technologies.
• Evaluate the current landscape of gene therapy research and its impact on future medical practices and societal norms.
  • The current landscape of gene therapy research is rapidly evolving, with advances in technologies like CRISPR enhancing our ability to modify genes accurately. As more therapies receive approval and show efficacy in treating previously untreatable conditions, this could significantly reshape medical practices by offering personalized medicine approaches tailored to an individual's genetic makeup. However, this evolution also invites scrutiny over societal norms regarding genetics—questions about equity in access to these therapies may challenge existing healthcare structures and necessitate discussions on moral responsibilities in medical innovation.

Related terms

CRISPR: A revolutionary technology that allows for precise editing of DNA within living organisms, making gene modification more accessible and efficient.

Genetic Disorder: A disease caused by abnormalities in an individual's DNA, which can be inherited or occur due to mutations.

Somatic vs. Germline Therapy: Somatic therapy targets non-reproductive cells and affects only the individual, while germline therapy involves changes to reproductive cells that can be passed on to future generations.