AANS Neurosurgeon | Volume 26, Number 3, 2017

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Unique Gene Therapy Prevents, Reverses Multiple Sclerosis in Animal Model

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

By combining a brain-protein gene and an existing medication, the researchers were able to prevent the mouse version of multiple sclerosis. Likewise, the treatments produced near-complete remission in the animal models. The findings, researchers said, have significant potential for treating multiple sclerosis and other autoimmune disorders.

Multiple sclerosis affects about 2.3 million people worldwide and is the most common neurological disease in young adults. The 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.

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