Multiple Sclerosis (MS) and its animal model, experimental autoimmune encephalitis

(EAE), are autoimmune disease of the central nervous system (CNS) characterized by

inflammation and demyelination. Neurological gene therapy strategies involving the use

of therapeutic transgenes expressed by viral vectors, DNA vectors, recombinant proteins

or myelin-expressing cells have shown indications of inhibiting autoimmune-mediated

inflammation in the brain. Here we review currently used pharmacological approaches

for treating MS and we describe the currently used gene and cell therapy approaches

for treating MS and his rodent model (EAE). Moreover, we summarize the advantage

and disadvantages of these methodology and clinical studies. Also, we will propose five

different avenues that could be pursued with the objective of developing gene therapy

to treat MS, e.g., The inhibition of leukocyte migration into the CNS by targeting the

blood-brain barrier; Reducing the production of anti-myelin antibodies; Expressing

immune-modulating molecules within the affected CNS; Target differentiation factors to

oligodendrocytes precursors to repair damaged myelin, and the abrogation of the myelin

specific T-cell tolerance in the thymus and in the periphery. This will enable us to

design specific and new therapeutic strategy for the treatment of autoimmune disease

such as MS.