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 either aim to replace defective genes, knock out harmful genes, or introduce new genes to help combat a disease. The distinction between using gene therapy for therapeutic purposes versus enhancement has become a critical debate, especially as new technologies emerge.

5 Must Know Facts For Your Next Test

1. Gene therapy can utilize various methods, including viral vectors to deliver therapeutic genes into a patient's cells.
2. The first approved gene therapy product in the U.S. was on the market in 2017, marking a significant advancement in the field.
3. Gene therapy holds potential for treating a range of conditions, including inherited disorders, some types of cancer, and certain viral infections.
4. There are ethical considerations surrounding gene therapy, particularly concerning enhancement versus treatment and the implications of modifying human genetics.
5. Clinical trials for gene therapy are ongoing, with researchers continuously exploring its safety and effectiveness across different diseases.

Review Questions

• What are the key differences between using gene therapy for therapeutic purposes versus enhancement?
  • Therapeutic gene therapy aims to correct or alleviate the symptoms of genetic disorders by fixing defective genes or providing functional copies of genes. In contrast, enhancement gene therapy seeks to improve or augment human traits beyond normal health levels, such as increasing physical abilities or cognitive functions. The ethical implications of these two approaches have sparked significant debate regarding the potential consequences of altering human genetics and the boundaries of acceptable medical practices.
• Discuss the significance of viral vectors in the delivery of gene therapy and the challenges they present.
  • Viral vectors are crucial in gene therapy as they facilitate the transfer of therapeutic genes into target cells. These vectors are engineered from viruses that can efficiently deliver genetic material into human cells while minimizing adverse effects. However, challenges arise concerning immune responses against the vectors, potential insertional mutagenesis where genes disrupt normal cell function, and ensuring long-term expression of the therapeutic genes. Overcoming these challenges is essential for making gene therapy safe and effective.
• Evaluate the ethical considerations related to germline gene therapy and its potential impact on future generations.
  • Germline gene therapy raises significant ethical questions because it involves making permanent changes to an individual's genetic code that can be inherited by future generations. This capability raises concerns about unintended consequences, such as unforeseen genetic defects or social inequalities created by access to enhancement technologies. Additionally, there is a fear that such interventions could lead to 'designer babies' where parents might choose traits for their children based on personal preferences rather than health needs. The long-term effects on humanity and natural evolution remain uncertain, which makes this area highly controversial within bioethics.

Related terms

Genetic Engineering: The process of altering the genetic material of an organism, which can include gene therapy, to create desired traits or to correct genetic disorders.

Somatic Cell Therapy: A type of gene therapy that targets somatic (non-reproductive) cells, aiming to treat diseases without affecting future generations.

Germline Gene Therapy: An experimental technique aimed at altering the genes in germline cells (sperm and egg) so that the changes are heritable and passed down to future generations.