Gene therapy

Definition

Gene therapy is a medical technique that involves modifying or manipulating genes to treat or prevent diseases, particularly genetic disorders. This approach can involve inserting new genes, repairing faulty ones, or altering the expression of existing genes to achieve a therapeutic effect. Gene therapy has the potential to address a wide range of diseases, including inherited conditions and certain types of cancer, and it often intersects with concepts like horizontal gene transfer.

5 Must Know Facts For Your Next Test

1. Gene therapy can be categorized into two main types: somatic gene therapy, which targets non-reproductive cells, and germline gene therapy, which affects reproductive cells and can be passed on to future generations.
2. One of the earliest successful applications of gene therapy was in treating severe combined immunodeficiency (SCID) by correcting a specific genetic defect in immune cells.
3. Gene therapy can utilize various vectors, including viruses and plasmids, to deliver therapeutic genes into target cells effectively.
4. Challenges in gene therapy include ensuring long-term expression of the introduced genes and avoiding immune responses against the vectors used for delivery.
5. Regulatory frameworks for gene therapy are critical as they ensure the safety and efficacy of treatments before they can be approved for clinical use.

Review Questions

• How does gene therapy utilize horizontal gene transfer mechanisms in its applications?
  • Gene therapy often employs principles similar to horizontal gene transfer, as it involves transferring genetic material between organisms or cells. By using vectors, such as viruses that naturally incorporate their genetic material into host genomes, gene therapy can mimic these natural processes to deliver therapeutic genes. Understanding horizontal gene transfer mechanisms aids in developing more effective vectors and improving the overall efficiency of gene delivery.
• Evaluate the ethical considerations surrounding germline gene therapy compared to somatic gene therapy.
  • Germline gene therapy raises significant ethical concerns because it involves altering genes in reproductive cells, which can affect not only the individual treated but also future generations. This creates debates about potential unintended consequences on the human gene pool and raises questions about 'designer babies' and eugenics. In contrast, somatic gene therapy targets only non-reproductive cells and does not pose risks to future offspring, making it more ethically acceptable within current societal norms. The distinction between these two approaches is crucial for ongoing discussions about the implications of genetic engineering.
• Analyze how advancements in gene therapy could reshape our understanding of genetic disorders and treatment strategies in the future.
  • Advancements in gene therapy have the potential to revolutionize our understanding of genetic disorders by providing precise molecular solutions rather than traditional symptomatic treatments. With techniques like CRISPR allowing for targeted editing of defective genes, we may see a shift from managing diseases to curing them at their source. This could lead to personalized medicine approaches where treatments are tailored to individual genetic profiles. The implications extend beyond individual health; as we gain more insights into genetic functions and interactions through gene therapy research, we could develop comprehensive strategies for disease prevention and health optimization across populations.

Related terms

CRISPR: A revolutionary gene-editing technology that allows scientists to precisely alter DNA sequences and modify gene function.

Vector: A delivery system used in gene therapy to transport therapeutic genes into the target cells, often utilizing modified viruses.

Transfection: The process of introducing nucleic acids into cells to produce genetically modified cells, often a step in gene therapy applications.