New Gene Therapy Could Reverse Paralysis in Multiple Sclerosis

These T-regulatory cells, called Tregs, play a critical role in immune tolerance. The function of Tregs is to suppress the effector CD4+ T cell subsets which play a role in controlling and mediating autoimmune responses. Tregs have been found to be part of the pathogenesis of MS. Good therapies work by controlling the disease through the selective induction of auto-antigen specific CD4+CD25+FOXP3+ (forkhead box P3) Tregs. Past research has indicated that Treg-based immunotherapies may be effective. In one study, researchers were able to prevent or reduce the effects of autoimmune encephalomyelitis (EAE) in mouse models through the transfer of polyclonal CD4+CD25+ Tregs. Challenges remain in developing an immune-regulatory therapy that is robust and sustainable.

The team of researchers from the University of Florida used adeno-associated virus (AAV) immunotherapy to reverse the degenerative effects of MS. The team of researchers developed the in-vivo gene therapy with the use of pre-clinical mouse models. The researchers designed a gene transfer vector that targets the liver and allows the expression of full-length myelin oligodendrocyte glycoprotein (MOG) inside of hepatocytes (liver cells). This novel gene immune therapy causes a production of MOG-specific Tregs that seem to restore the immune response in patients with MS.