Gene therapy

Definition

Gene therapy is a medical technique that involves altering the genes inside a person's cells to treat or prevent disease. It aims to correct defective genes responsible for disease development, providing potential cures for genetic disorders. By utilizing various methods, such as inserting healthy genes, repairing mutated genes, or disabling problematic genes, gene therapy is closely linked to advancements in genetic engineering and the Human Genome Project, which maps the entire human genome and highlights genetic variations associated with diseases.

5 Must Know Facts For Your Next Test

1. Gene therapy has shown promise in treating genetic disorders such as cystic fibrosis, hemophilia, and certain types of muscular dystrophy.
2. The Human Genome Project has provided a comprehensive map of human genes, which has facilitated the identification of genetic mutations that cause diseases.
3. There are two main types of gene therapy: in vivo, where the gene is delivered directly into the patient’s body, and ex vivo, where cells are modified outside the body before being reintroduced.
4. Clinical trials have been conducted for gene therapies targeting inherited diseases and some types of cancers, leading to FDA approvals for specific treatments.
5. Ethical considerations surround gene therapy, including concerns about safety, long-term effects, and the potential for designer babies.

Review Questions

• How does gene therapy utilize information from the Human Genome Project to develop treatments for genetic disorders?
  • Gene therapy relies heavily on the data provided by the Human Genome Project to identify specific genes and mutations associated with various genetic disorders. By mapping out the human genome, researchers can pinpoint exactly where defects occur within an individual's DNA. This knowledge allows scientists to design targeted therapies that can correct or compensate for these defective genes, thus providing potential treatments for conditions like cystic fibrosis or hemophilia.
• What are the major ethical concerns related to gene therapy that researchers must consider when developing new treatments?
  • Ethical concerns surrounding gene therapy primarily focus on safety, long-term effects, and potential misuse. There is a fear that altering genes may lead to unforeseen consequences or adverse health effects over time. Additionally, the possibility of creating 'designer babies' raises questions about societal implications and equity in access to such technologies. Researchers must navigate these ethical dilemmas carefully while advancing scientific understanding and ensuring patient safety.
• Evaluate the impact of CRISPR technology on the future of gene therapy and its potential to revolutionize treatment options for genetic disorders.
  • CRISPR technology has dramatically transformed the landscape of gene therapy by offering a more precise and efficient method for editing genes. This innovation allows scientists to make specific changes at targeted locations in the genome quickly and cost-effectively. As CRISPR continues to be refined and its applications expand, it holds the potential to revolutionize treatment options for a range of genetic disorders. However, challenges regarding ethical considerations, off-target effects, and regulatory frameworks will need to be addressed as this technology advances toward clinical use.

Related terms

CRISPR: A revolutionary gene-editing technology that allows for precise modifications of DNA, enabling scientists to add, remove, or alter genetic material within organisms.

Somatic Cells: Any cells of the body excluding sperm and egg cells; gene therapy often targets somatic cells to treat diseases without affecting future generations.

Vector: A delivery system used in gene therapy to transport therapeutic genes into target cells; commonly used vectors include viruses that have been modified to be safe for human use.