Kymriah is made by taking a person’s own T cells (a type of white blood cell), and modifying them in a laboratory so they contain a specific protein. Now consider Kymriah (tisagenlecleucel), a gene therapy used to treat acute lymphoblastic leukemia and certain types of non-Hodgkin’s lymphoma. When Luxturna is injected in the eye, this healthy copy acts in place of the mutated RPE65 gene and restores vision loss. Luxturna contains a healthy copy of the RPE65 gene, a gene that can lead to blindness if mutated. Let’s talk about a couple examples of gene therapy so you can see how they work in real life.Ĭonsider Luxturna (voretigene neparvovec), a type of gene therapy used to treat a type of inherited vision loss. Killing disease-causing cells: giving an unhealthy cell “instructions” (a set of new DNA) which causes the cell to die
Gene augmentation: adding a health copy of a gene to a cell where a faulty gene exists, so the healthy copy can override the negative effects caused by the faulty gene Gene inhibition: turning off the gene that is responsible for encouraging disease The goal of gene therapy is to change how existing genes in your body operate. They are called “biologics” because they come from living sources, not chemicals. Gene therapy is considered a type of biologic treatment. Gene therapy is a type of treatment used to treat genetic disorders and other medical conditions. In this article, we’ll talk about the pros and cons of gene therapy. A genetic disorder is a medical condition caused by DNA that is different from what is considered normal. Over three decades later, we’re still learning about its true potential in treating various medical conditions, including genetic disorders and cancer. The first gene therapy study was conducted in 1990.
It also could mean an entirely new gene is introduced to the body to help treat the medical condition. Or, it could mean making the faulty gene inactive. This could mean replacing the faulty gene with a healthy copy. The goal is to return any abnormal DNA to normal conditions. Gene therapy gives us a way to modify our DNA. Other times, they’re caused by something in the environment, such as frequent sun exposure. Sometimes these are inherited, which means they’re passed from parent to child. But, sometimes this DNA sequence is different than usual. Every unique gene has a DNA sequence that is considered “normal” for that particular gene. Our genes play a big role in our overall health. You’re the only person on the planet who has your exact DNA (unless you’re an identical twin!) They live in almost every cell in your body, and they’re made up of a chemical called DNA. You can think of genes as the body’s “instruction manual.” Your genes tell your body how to work. Have you ever wondered what makes you, well, you? Your eye color, the texture of your hair, and even aspects of your personality are all determined by your genes.
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