Gene therapy

Definition

Gene therapy is a medical technique that modifies a person's genes to treat or prevent disease, often by correcting defective genes or introducing new ones. This approach can target various conditions, including genetic disorders, some cancers, and certain viral infections, thereby transforming how these diseases are managed. The underlying goal of gene therapy is to restore normal function in affected cells and tissues, making it a powerful tool in modern medicine.

5 Must Know Facts For Your Next Test

1. Gene therapy can potentially cure genetic disorders by correcting the underlying genetic defects responsible for the disease.
2. It can be performed in vivo (directly in the body) or ex vivo (cells are modified outside the body and then returned).
3. Clinical trials have shown promise in using gene therapy to treat conditions like cystic fibrosis, hemophilia, and certain types of muscular dystrophy.
4. There are ethical considerations regarding gene therapy, especially concerning germline modifications that could be passed on to future generations.
5. While still largely experimental, gene therapy has already seen successful applications in some patients, leading to significant improvements in their health outcomes.

Review Questions

• How does gene therapy differ from traditional treatment methods for genetic disorders?
  • Gene therapy differs from traditional treatment methods as it targets the root cause of genetic disorders by modifying or replacing defective genes, rather than just managing symptoms. Traditional methods may involve medication or surgeries aimed at alleviating symptoms without addressing the underlying genetic issue. By directly altering the genetic material, gene therapy holds the potential for a more permanent solution to conditions that have previously been considered chronic or incurable.
• Discuss the role of viral vectors in gene therapy and their importance in delivering therapeutic genes.
  • Viral vectors play a crucial role in gene therapy by serving as vehicles to deliver therapeutic genes into target cells. These vectors are modified viruses that can efficiently enter cells and transport genetic material without causing disease. Their ability to integrate into host genomes allows for stable expression of the introduced genes, making them essential for achieving long-lasting effects in treating genetic disorders. Understanding how to design and utilize these vectors safely is key to successful gene therapy applications.
• Evaluate the ethical implications of gene therapy, particularly regarding germline modifications and their potential impact on future generations.
  • The ethical implications of gene therapy are significant, especially when considering germline modifications that can be inherited by future generations. This raises concerns about unintended consequences on human evolution, societal inequality regarding access to such therapies, and potential 'designer babies' where traits could be chosen based on personal or societal preferences. A thorough evaluation of these issues is necessary as the technology advances, requiring a balance between innovation and moral responsibility to ensure it benefits all of humanity without compromising ethical standards.

Related terms

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

viral vectors: Tools commonly used to deliver genetic material into cells, often derived from viruses that have been modified to be safe for therapeutic use.

somatic cell therapy: A type of gene therapy that targets non-reproductive cells in the body, focusing on treating specific tissues or organs rather than altering the germline.