Gene therapy

Definition

Gene therapy is a medical technique that involves the modification or manipulation of genes to treat or prevent disease. It aims to correct defective genes responsible for disease development, potentially offering a permanent solution rather than just alleviating symptoms. By introducing, removing, or altering genetic material within a person's cells, gene therapy can address issues related to protein misfolding and associated diseases, which often stem from genetic mutations.

5 Must Know Facts For Your Next Test

1. Gene therapy can be categorized into two main types: somatic cell gene therapy, which affects non-reproductive cells, and germline gene therapy, which targets reproductive cells and can be inherited by future generations.
2. One significant challenge in gene therapy is delivering the therapeutic gene effectively to the right cells without triggering an immune response.
3. Gene therapy has shown promise in treating various conditions, including certain types of inherited blindness and muscular dystrophy.
4. Researchers are also exploring gene therapy as a potential treatment for protein misfolding diseases, like cystic fibrosis and Alzheimer's disease, by targeting the genes responsible for producing malfunctioning proteins.
5. Ethical considerations surrounding gene therapy include concerns about its long-term effects, potential misuse in 'designer babies', and access disparities in healthcare.

Review Questions

• How does gene therapy relate to the treatment of diseases caused by protein misfolding?
  • Gene therapy directly addresses diseases caused by protein misfolding by targeting the genes responsible for producing these defective proteins. By correcting or replacing faulty genes, gene therapy aims to restore normal protein function and alleviate symptoms associated with these disorders. This approach can offer a more permanent solution than traditional treatments that only manage symptoms without addressing the root cause of the disease.
• Discuss the role of vectors in gene therapy and their significance in treating genetic disorders.
  • Vectors are crucial in gene therapy as they serve as delivery systems to transport therapeutic genes into target cells. Commonly modified viruses are used as vectors because they can efficiently introduce genetic material into human cells. The success of gene therapy largely depends on the effectiveness of these vectors to ensure that the correct cells receive the therapeutic gene while minimizing adverse immune responses and ensuring safety.
• Evaluate the ethical implications of using gene therapy in the context of protein misfolding diseases and broader healthcare practices.
  • The use of gene therapy raises significant ethical implications, especially concerning its application in treating protein misfolding diseases. While it offers hope for curing conditions previously deemed untreatable, concerns about long-term effects and potential misuse—such as creating 'designer babies'—must be carefully considered. Additionally, equitable access to these advanced therapies poses a challenge, as disparities in healthcare could result in unequal benefits from such innovative treatments. Addressing these ethical concerns is essential for responsible progression in the field of gene therapy.

Related terms

CRISPR: A revolutionary gene-editing technology that allows for precise alterations in DNA, enabling researchers to modify genes easily and efficiently.

Vector: A delivery system used in gene therapy to transport the therapeutic gene into the patient's cells, often utilizing viruses that have been modified to be safe.

Monogenic disorders: Diseases caused by a mutation in a single gene, which can be targeted by gene therapy to correct the underlying genetic defect.