Gene therapy

Definition

Gene therapy is a medical technique that involves altering or manipulating genes within an individual's cells to treat or prevent disease. This innovative approach often aims to replace, inactivate, or introduce genes into a patient's cells to correct genetic disorders, potentially offering a long-term solution rather than merely alleviating symptoms. It connects deeply with various genetic engineering techniques and has significant implications for applications in biotechnology, particularly in treating inherited diseases and certain types of cancer.

5 Must Know Facts For Your Next Test

1. Gene therapy has the potential to treat a wide range of diseases, including genetic disorders, some forms of cancer, and certain viral infections.
2. It can involve various methods such as replacing a missing or nonfunctional gene, repairing a mutated gene, or introducing a new gene to help fight disease.
3. The first approved gene therapy product for human use was Glybera, which was approved in Europe in 2012 for treating a rare genetic disorder called lipoprotein lipase deficiency.
4. Challenges in gene therapy include potential immune responses to the delivery vectors and ensuring that the therapeutic genes are expressed at the right levels and in the right cells.
5. Clinical trials are ongoing to explore gene therapies for conditions like cystic fibrosis, muscular dystrophy, and sickle cell disease, showing promise for future treatment options.

Review Questions

• How does gene therapy utilize genetic engineering techniques to address genetic disorders?
  • Gene therapy uses genetic engineering techniques such as CRISPR and vectors to directly target and modify specific genes associated with genetic disorders. By employing these methods, scientists can either replace defective genes with functional ones or introduce new genes that produce beneficial effects. This approach not only aims at correcting the underlying cause of the disease but also highlights how advancements in genetic engineering can pave the way for innovative treatment options.
• Discuss the potential ethical concerns surrounding gene therapy and its applications in biotechnology.
  • The use of gene therapy raises several ethical concerns, especially regarding its application in germline editing, where changes could be passed down to future generations. Issues include the risk of unintended consequences from modifying genes, potential socioeconomic disparities in access to these therapies, and the moral implications of 'designer babies' where parents might choose traits for their children. Addressing these concerns requires careful consideration of regulatory frameworks and public discourse surrounding genetic technologies.
• Evaluate the impact of ongoing clinical trials for gene therapies on future healthcare practices and patient outcomes.
  • Ongoing clinical trials for gene therapies are likely to have a significant impact on future healthcare practices by providing insights into effective treatments for previously untreatable genetic conditions. As more successful therapies emerge from these trials, it could lead to widespread adoption of personalized medicine approaches that focus on individual genetic profiles. The successes observed in clinical trials could also shift the landscape of healthcare towards more preventive measures rather than solely reactive treatments, ultimately improving patient outcomes and quality of life.

Related terms

CRISPR: A powerful gene-editing tool that allows scientists to easily alter DNA sequences and modify gene function, enhancing the possibilities for gene therapy.

Vectors: Delivery systems used in gene therapy to transfer therapeutic genes into patients' cells, commonly using modified viruses to carry the new genetic material.

Monogenic disorders: Genetic diseases caused by mutations in a single gene, often prime targets for gene therapy approaches aiming to correct these specific mutations.