Genetic Therapies Treatments

Blood and immune conditions

If you have a blood or immune condition, the doctor may take blood from your veins or bone marrow from your hip bone to be modified in a laboratory with genetic therapy. Blood and bone marrow contain hematopoietic stem cells , which produce other key cells that make up the blood and immune system.

In the laboratory, scientists may use either gene transfer or genome editing to change the sample of stem cells provided by you. The modified cells are returned to your body through an intravenous (IV) line in one of your blood vessels. This procedure is called cell-based therapy or a blood and bone marrow transplant. The altered hematopoietic stem cells will produce healthy blood or immune cells.

Illustration of cell-based genetic therapy — **Cell-based genetic therapy.** The image shows how doctors take a patient’s hematopoietic stem cells to modify with genetic approaches in the laboratory. After the cells are modified, they go back into the patient’s body through a blood and bone marrow transplant. Medical Illustration Copyright © 2022 Nucleus Medical Media, All rights reserved.

Genetic therapies for sickle cell disease are examples of how this approach is being developed. Currently, a blood and bone marrow transplant of stem cells from a relative or an unrelated well-matched donor can cure sickle cell disease, but many patients do not have a well-matched donor available. Genetic therapies that modify a person’s own hematopoietic stem cells may provide a cure for people who do not have a well-matched donor. Modified hematopoietic stem cells can be injected into the blood, then the cells travel in the bloodstream to the marrow spaces inside the bones. Once inside the bone marrow, the cells can produce healthy red blood cells that do not sickle. Watch the video below to learn more about how genome editing is being tested for sickle cell disease.

Treatments for organs or tissues

Sometimes, genetic therapies are needed to alter cells that cannot be easily removed from the patient for treatment in the laboratory. Researchers are still working on ways to deliver the genetic therapy for these conditions. The methods being studied include IV infusion into the bloodstream, injection directly into an organ, and other ways to directly deliver the therapy into the affected tissues.

One condition that could be treated this way is hemophilia B. Hemophilia is caused by a faulty gene that prevents production of a protein necessary to clot the blood after an injury. Genetic therapy that is being developed for Hemophilia B involves infusing a vector carrying the normal clotting factor gene into the bloodstream. The vector then reaches liver cells to produce the clotting factor needed for better health.

Clinical Trials

We lead or sponsor many studies relevant to genetic therapies using gene transfer or genome editing. See whether you or someone you know is eligible to participate in our clinical trials.

Learn more about participating in a clinical trial.