Gene Therapy to Prevent the Progression of Multiple Sclerosis (MS)

Technology #15158

Treatment Targets the Liver to Restore Immune Tolerance and Reduce Autoimmune Inflammation

This gene therapy treatment re-establishes immune tolerance in order to halt the progression of multiple sclerosis, a debilitating disease that affects more than 2.3 million people worldwide. Adeno-associated virus gene therapy (AAV therapy) has been proven as a powerful tool for the treatment of a broad spectrum of diseases. Researchers at the University of Florida have developed the first AAV therapy that treats autoimmune diseases by exploiting the liver’s natural tolerogenic ability to generate cells that suppress harmful autoimmune responses. Multiple sclerosis is an immune-mediated disease of the central nervous system (CNS) in which a patient’s immune system loses its ability to distinguish between self and attacker. The dysfunctional immune system attacks myelin, an insulating sheath that protects neuron signaling speed, causing demyelination and neuronal degeneration. It’s estimated that more than 400,000 people in the United States have MS, with about 200 new cases being diagnosed each week.


Multiple sclerosis gene therapy


  • Allows for greater immune control and stabilization, preventing and halting the progression of multiple sclerosis
  • Establishes self-sustained immune tolerance, reducing need for long-term therapies and increasing patient comfort and ease of treatment
  • Creates potential for a new line of MS treatment, advancing MS and gene therapy research
  • Enables the patient’s own immune system to generate the unique immune regulatory T-Cells specifically needed, permitting a universally applicable treatment


Regulatory T-Cells are critical to the maintenance of immune self-tolerance and homeostasis; they act as a self-check built into the immune system to prevent the excessive and self-destructive immune responses found in multiple sclerosis patients. Researchers at the University of Florida have developed an AAV gene therapy that allows the body to manufacture the specific regulatory T-cells it needs. This helps the body re-establish immune tolerance and provides a universally applicable treatment. The self-sustaining nature of the treatment may eliminate the need for long-term treatments. The AAV gene therapy treatment is directed at the liver, an organ with an unusual capacity to induce immune tolerance. Several proteins are expressed, which unlike current therapies, would create a treatment that is persistent. Re-establishing the body’s immune tolerance would both prevent and halt the existing demyelinating disease, due to the greater immune control and stabilization.