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.
ApplicationMultiple 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