What is gene therapy?
Q: What is gene therapy?
A: Gene therapy is the process of inserting a working gene to replace a damaged gene in the patient.
Q: What product has the European Commission approved for gene therapy?
A: The European Commission has approved the product Glybera for gene therapy.
Q: How does Glybera treat the genetic disorder?
A: Glybera uses a virus to infect muscle cells with a working copy of the gene to treat the genetic disorder.
Q: What is the lipase gene, and why is it important?
A: The lipase gene is a gene needed to break down fats. It is important because having damaged copies of the gene can cause fat to build up in the blood, leading to pancreatitis, which is life-threatening.
Q: What technique does Glybera use for gene therapy?
A: Glybera uses the transfection technique, where the viral vector sticks the gene into human cells.
Q: What is a viral vector?
A: A viral vector is a carrier that carries genetic material into cells.
Q: How is the transfection technique different from the gene knockout technique?
A: The transfection technique involves using a viral vector to insert a working copy of the gene, whereas the gene knockout technique does not use a viral vector. The goal of the gene knockout technique is to remove or disable a gene.
