Gene therapy

Definition

Gene therapy is a medical technique that aims to treat or prevent disease by introducing, removing, or altering genetic material within a patient's cells. This approach can potentially correct genetic disorders at their source and has applications that span various fields, including medicine, biotechnology, and genetics.

5 Must Know Facts For Your Next Test

1. Gene therapy can be delivered through several methods, including direct injection of therapeutic DNA, the use of viral vectors, or through CRISPR-based techniques.
2. One key goal of gene therapy is to replace or repair defective genes that cause disease, which can lead to long-term benefits for patients.
3. Current research in gene therapy focuses on treating genetic disorders such as cystic fibrosis, muscular dystrophy, and certain types of cancer.
4. Regulatory agencies are closely monitoring gene therapy treatments due to ethical concerns and the potential for unintended consequences in the genome.
5. Clinical trials are essential for advancing gene therapy, helping to assess its safety, efficacy, and potential long-term effects on patients.

Review Questions

• How does gene therapy utilize genome editing technologies to address genetic disorders?
  • Gene therapy often employs genome editing technologies like CRISPR to precisely target and modify faulty genes associated with genetic disorders. By correcting these genes, gene therapy aims to alleviate or even cure conditions that arise from genetic defects. This integration of cutting-edge genome editing into gene therapy practices enhances the ability to provide personalized and effective treatments for patients.
• What role do viral vectors play in the delivery of gene therapy treatments?
  • Viral vectors are crucial in gene therapy as they serve as vehicles to transport therapeutic genes into target cells. These engineered viruses can effectively penetrate cell membranes and deliver the intended genetic material directly into the cells' nuclei. The use of viral vectors enhances the efficiency of gene delivery, making it easier for the body to incorporate new genetic information necessary for treating various diseases.
• Evaluate the ethical considerations surrounding gene therapy and its implications for future treatments.
  • The ethical considerations surrounding gene therapy include concerns about safety, informed consent, and the potential for unintended genetic alterations. As gene therapy continues to evolve, issues such as accessibility, equity in healthcare, and the possibility of 'designer babies' raise significant moral questions. Evaluating these concerns is vital for ensuring responsible advancement in gene therapy that prioritizes patient welfare and societal impact while fostering innovation in treatment options.

Related terms

CRISPR:

A revolutionary genome editing technology that allows for precise alterations to DNA sequences, enabling targeted modifications in genes.

viral vectors: Engineered viruses used to deliver therapeutic genes into cells as part of gene therapy strategies.

somatic cell therapy: A type of gene therapy that involves modifying the genes in somatic (non-reproductive) cells to treat a disease.