![]() The cells are later injected into the patient, where the new gene is used to produce the desired effect. The vector containing the desired gene is introduced into these cells. ![]() When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab. Gene therapy can be used to modify cells inside or outside the body. When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient. Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease. Viruses are currently the most commonly used vectors in gene therapies because they have a natural ability to deliver genetic material into cells. Vectors need to be able to efficiently deliver genetic material into cells, and there are different kinds of vectors. To insert new genes directly into cells, scientists use a vehicle called a “vector.” Vectors are genetically engineered to deliver the necessary genes for treating the disease. Or they can turn off genes that are causing problems.They can add genes to the body to help treat disease.They can replace a gene that is missing or is causing a problem.But some inherited and acquired mutations can cause developmental disorders, neurological diseases, and cancer.ĭepending on what is wrong, scientists can do one of several things in gene therapy: The good news is that most of these genetic changes (mutations) do not cause disease. These acquired mutations can be caused by environmental exposures. In addition, healthy genes can change (mutate) over the course of our lives. This is typically referred to as a genetically inherited mutation. Sometimes the whole or part of a gene is defective or missing from birth. The tissues and organs in turn support all our body’s functions. Groups of many cells make up the tissues and organs of the body, including muscles, bones, and blood. The genes provide the information that makes different cells do different things. Cells are the basic building blocks of all living things the human body is composed of trillions of them. ![]() Within the cells of our bodies, there are thousands of genes that provide the information to produce specific proteins that help make up the cells. Food and Drug Administration has approved multiple gene therapy products for cancer and rare disease indications. This research is paying off, as advancements in science and technology today are changing the way we define disease, develop drugs, and prescribe treatments. Recognizing this, scientists have worked for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure, or prevent a disease or medical condition. Indeed, a defective gene or genes can make you sick. The genes in your body’s cells play a key role in your health.
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