ABSTRACT
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.