Multiple Sclerosis (MS) is the most common autoimmune demyelinating disease of the
Central Nervous System (CNS). It is a multifactorial disease which develops in an immune-mediated
way under the influences of both genetic and environmental factors. Demyelination is observed in the
brain and spinal cord leading to neuro-axonal damage in patients with MS. Due to the infiltration of
different immune cells such as T-cells, B-cells, monocytes and macrophages, focal lesions are observed
in MS. Currently available medications treating MS are mainly based on two strategies; i) to
ease specific symptoms or ii) to reduce disease progression. However, these medications tend to induce
different adverse effects with limited therapeutic efficacy due to the protective function of the
blood-brain barrier. Therefore, researchers have been working for the last four decades to discover
better solutions by introducing gene therapy approaches in treating MS generally by following three
strategies, i) prevention of specific symptoms, ii) halt or reverse disease progression and iii) heal CNS
damage by promoting remyelination and axonal repair. In last two decades, there have been some remarkable
successes of gene therapy approaches on the experimental mice model of MS - experimental
autoimmune encephalomyelitis (EAE) which suggests that it is not far that the gene therapy approaches
would start in human subjects ensuring the highest levels of safety and efficacy. In this review, we
summarised the gene therapy approaches attempted in different animal models towards treating MS.
Keywords: Gene therapy, multiple sclerosis, autoimmune disease, autoimmunity, demyelination, experimental autoimmune
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