Muscular dystrophy Is It Safe? Safety is one of the top priorities in clinical trials. In the case of gene therapy, these studies clearly helped.
Gene Therapy Successes Gene Therapy Successes Researchers have been working for decades to bring gene therapy to the clinic, yet very few patients have received any effective gene-therapy treatments. Even though gene therapy has been slow to reach patients, its future is very encouraging.
Decades of research have taught us a lot about designing safe and effective vectors, targeting different types of cells, and managing and minimizing immune responses in patients.
Today, many clinical trials are underway, where researchers are carefully testing treatments to ensure that any gene therapy brought into the clinic is both safe and effective. Below are some gene therapy success stories.
Successes represent a variety of approaches—different vectors, different target cell populations, and both in vivo and ex vivo approaches—to treating a variety of disorders.
Sebastian Misztal was a patient in a hemophilia gene therapy trial in Following the treatment, Misztal no longer had spontaneous bleeding episodes. Most commonly, blood stem cells are removed from patients, and retroviruses are used to deliver working copies of the defective genes.
After the genes have been delivered, the stem cells are returned to the patient.
Severe Combined Immune Deficiency SCID was one of the first genetic disorders to be treated successfully with gene therapy, proving that the approach could work. However, the first clinical trials ended when the viral vector triggered leukemia a type of blood cancer in some patients. Since then, researchers have begun trials with new, safer viral vectors that are much less likely to cause cancer.
Adenosine deaminase ADA deficiency is another inherited immune disorder that has been successfully treated with gene therapy. For the majority of patients in these trials, immune function improved to the point that they no longer needed injections of ADA enzyme.
Importantly, none of them developed leukemia. Hereditary blindness Gene therapies are being developed to treat several different types of inherited blindness—especially degenerative forms, where patients gradually lose the light-sensing cells in their eyes. Encouraging results from animal models especially mouse, rat, and dog show that gene therapy has the potential to slow or even reverse vision loss.
The eye turns out to be a convenient compartment for gene therapy. The retina, on the inside of the eye, is both easy to access and partially protected from the immune system.
Most gene-therapy vectors used in the eye are based on AAV adeno-associated virus. In one small trial of patients with a form of degenerative blindness called LCA Leber congenital amaurosisgene therapy greatly improved vision for at least a few years.
However, the treatment did not stop the retina from continuing to degenerate. In another trial, 6 out of 9 patients with the degenerative disease choroideremia had improved vision after a virus was used to deliver a functional REP1 gene.
Hemophilia People with hemophilia are missing proteins that help their blood form clots. Those with the most-severe forms of the disease can lose large amounts of blood through internal bleeding or even a minor cut.
In a small trial, researchers successfully used an adeno-associated viral vector to deliver a gene for Factor IX, the missing clotting protein, to liver cells. After treatment, most of the patients made at least some Factor IX, and they had fewer bleeding incidents.
Blood disease Patients with beta-Thalassemia have a defect in the beta-globin gene, which codes for an oxygen-carrying protein in red blood cells.
Many who have this disorder depend on blood transfusions for survival. Ina patient received gene therapy for severe beta-Thalassemia. Blood stem cells were taken from his bone marrow and treated with a retrovirus to transfer a working copy of the beta-globin gene.
The modified stem cells were returned to his body, where they gave rise to healthy red blood cells. Seven years after the procedure, he was still doing well without blood transfusions.
A similar approach could be used to treat patients with sickle cell disease. Fat metabolism disorder InGlybera became the first viral gene-therapy treatment to be approved in Europe.
The treatment uses an adeno-associated virus to deliver a working copy of the LPL lipoprotein lipase gene to muscle cells. The LPL gene codes for a protein that helps break down fats in the blood, preventing fat concentrations from rising to toxic levels.
Cancer Several promising gene-therapy treatments are under development for cancer. One, a modified version of the herpes simplex 1 virus which normally causes cold sores has been shown to be effective against melanoma a skin cancer that has spread throughout the body.ALS is a progressive disease, which means it gets worse over time.
It affects nerves in your brain and spinal cord that control your muscles. As your muscles get weaker, it gets harder for you to. Gene therapy carries the promise of cures for many diseases and for types of medical treatment that didn't seem possible until recently.
With its potential to eliminate and prevent hereditary diseases such as cystic fibrosis and hemophilia and its use as a possible cure for heart disease, AIDS, and.
In theory, there’s no limit to the types of diseases that could be treated with stem cell research. Given that researchers may be able to study all cell types via embryonic stem cells, they have the potential to make breakthroughs in any disease. Hemophilia is a rare bleeding disorder in which the blood does not clot normally, causing bleeding that can damage organs and tissues.
Learn more about causes, signs and symptoms, complications, diagnoses, treatments, and how to participate in clinical trials.
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