Gene therapy of multiple sclerosis

Abstract

Multiple sclerosis (MS) constitutes a difficult challenge for the design of innovative therapies: the aetiology is unknown, the pathogenesis only partially understood, and the whole process is multi-focal, chronic, and occurring beyond anatomical barriers, making the delivery of potentially therapeutic molecules difficult. Gene therapy, thus, constitutes a realistic alternative to ensure prolonged, and site-specific delivery of therapies. Recent advancements in the comprehension of the immunopathological processes leading to central nervous system inflammation, and the development of new gene therapy tools, such as RNA-interference, are rapidly leading to a large array of possibilities of intervention, documented in the present review in its animal model, experimental autoimmune encephalomyelitis (EAE). Since progressive forms of MS remain orphan of efficient therapies, the field is open for less conventional interventions such as gene therapy.