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 patient's cells. This innovative approach can address genetic disorders at their root cause, offering potential cures for inherited diseases and improving the effectiveness of existing treatments.

5 Must Know Facts For Your Next Test

1. Gene therapy can be categorized into two main types: somatic gene therapy, which modifies non-reproductive cells, and germline gene therapy, which affects reproductive cells and can be inherited by future generations.
2. One of the most significant applications of gene therapy is in treating genetic disorders like cystic fibrosis, muscular dystrophy, and hemophilia by correcting or replacing defective genes.
3. Recombinant DNA technology plays a crucial role in developing gene therapies by enabling scientists to manipulate genes and create vectors for delivering therapeutic genes to target cells.
4. The success of gene therapy relies heavily on the efficient delivery of the therapeutic genes, which is typically achieved using viral vectors due to their ability to penetrate host cells.
5. There are ongoing ethical debates surrounding gene therapy, particularly concerning germline modifications, as these could have long-term consequences for future generations and raise concerns about 'designer babies.'

Review Questions

• How does gene therapy utilize recombinant DNA technology in the treatment of genetic disorders?
  • Gene therapy employs recombinant DNA technology to create vectors that carry therapeutic genes into the patient's cells. This process involves manipulating DNA sequences to insert corrected versions of defective genes. By delivering these corrected genes directly to affected cells, gene therapy has the potential to address the underlying cause of genetic disorders rather than just treating symptoms.
• Discuss the differences between somatic and germline gene therapy and their implications for treatment options.
  • Somatic gene therapy targets non-reproductive cells and aims to treat specific conditions in an individual without affecting their offspring. In contrast, germline gene therapy alters reproductive cells, which means any changes made can be inherited by future generations. While somatic therapy is widely accepted and currently in use, germline therapy raises ethical concerns about potential long-term effects on the human gene pool and issues related to consent for future generations.
• Evaluate the ethical considerations involved in the use of gene therapy, particularly regarding germline modifications and their impact on society.
  • The ethical considerations surrounding gene therapy include concerns about safety, equity in access to treatments, and potential misuse of technology. Germline modifications pose unique challenges as they can lead to unintended consequences in the human gene pool. The prospect of 'designer babies' raises questions about societal values and the definition of normalcy. As advancements continue, it is essential for regulatory frameworks and public discourse to evolve alongside scientific developments to ensure responsible use of gene therapy.

Related terms

CRISPR-Cas9: A revolutionary genome-editing technology that allows for precise modifications to DNA, enabling targeted changes in genes and offering new avenues for gene therapy.

Vector: A delivery system used in gene therapy to carry therapeutic genes into the patient's cells, often derived from viruses that can efficiently enter human cells.

Somatic Cell Therapy: A type of gene therapy that targets non-reproductive cells, focusing on treating specific tissues or organs without affecting the germline.