Unique gene therapy prevents, reverses multiple sclerosis in animal model

Multiple sclerosis can be inhibited or reversed in mouse models using a novel gene therapy technique to suppress the immune response that induces the disease, University of Florida Health researchers have found.

By combining the transfer of a brain-protein gene with a drug used in organ transplant recipients, the researchers essentially cured mice of multiple sclerosis, resulting in near-complete remission of disease. Their findings, which the researchers said have significant potential for treating multiple sclerosis and other autoimmune disorders, are published today (Sept. 21) in the journal Molecular Therapy.

Multiple sclerosis affects about 2.3 million people worldwide and is the most common neurological disease in young adults. This incurable disorder starts when the immune system attacks the myelin sheath surrounding nerve fibers, making them misfire and leading to problems with muscle weakness, vision, speech and muscle coordination.

The researchers used a harmless virus, known as an adeno-associated virus or AAV, to deliver a gene coding one of the common immune targets, a myelin sheath protein called myelin oligodendrocyte glycoprotein or MOG, into the livers of the mouse models. The protein leads to the production of so-called regulatory T cells, which suppress the rogue immune system cells responsible for attacking the protective layer of nerve cells that defines multiple sclerosis. The effectiveness of this approach is based on targeting the gene therapy to the liver, which promotes immune tolerance.

“Using a clinically tested gene therapy platform, we are able to induce very specific regulatory T cells that target the self-reactive cells responsible for causing multiple sclerosis,” said Brad E. Hoffman, Ph.D., an associate professor in the departments of pediatrics and neuroscience at the University of Florida College of Medicine.

The gene therapy-induced protein, MOG, was found to be effective in preventing and reversing multiple sclerosis on its own and the results have been validated through multiple independent experiments. Overall, the therapy was tested in groups of five to 10 mice and the results were reproduced multiple times.  

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