Unique gene therapy found to reverse multiple sclerosis in mice

Multiple sclerosis is a neurological disease that disables the immune system and causes problems with the central nervous system. This degenerative disease is commonly found in young adults and is known to affect muscle, vision, and speech coordination. It is believed that there’s no cure for multiple sclerosis but treatment can help recovery from attacks and manage symptoms. However, a new gene therapy technique has been tested in animal models and has successfully reversed multiple sclerosis. 

Researchers from the University of Florida Health were able to inhibit the disease's immune response in mice. The findings are published in the journal Molecular Therapy. Researchers used a harmless virus known as adeno-associated virus to deliver a gene responsible for a brain protein into the livers of the mice. The virus caused production of T cells that suppress the immune system attack in multiple sclerosis. Brad E. Hoffman, Ph.D., assistant professor in the departments of pediatrics and neuroscience at the University of Florida College of Medicine, said: "Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis."

The treatment's longevity is especially noteworthy. The mouse models that were treated with gene therapy showed no signs of disease even after seven months. There is a need of further research involving other preclinical models before the therapy can be tested in humans during a clinical trial. Overall, researchers are confident that gene therapy can be effective in humans. 

DOI: 10.1016/j.ymthe.2017.09.001