Continuous identification of specific targets and candidate genes together with novel approaches offers new promises for the future of gene therapy design in Behçet's disease (BD). Personalized medicine based on pharmacogenomics is being developed at the clinical stage to improve treatment response. Screening the whole gene and regulatory regions is important when searching for novel variants associated with such complex diseases. Different host genetic factors play significant roles in susceptibility to BD. Thus, identifying these genes responsible for susceptibility and resistance to BD may offer a notable contribution toward understanding its pathogenesis, and may lead to the development of novel prophylactic and treatment strategies. Evidenced-based treatment strategy is recommended for the management in BD patients. This review sheds light on the immunopathogenesis and pharmacogenetics of BD with special attention to the treatment targeting gene polymorphisms. In conclusion, the potential of genetically guided treatment in BD takes us back to the future for an accurate management strategy of this serious rheumatic disease. The ongoing discovery of pivotal genes related to the susceptibility, manifestations, disease activity and treatment options provide substantial hope to the reduced frequency of BD, effective control and improvement in the prognosis. Targeted gene therapy could be a leading option in the treatment armamentarium of BD.
Keywords: Behçet's disease; gene therapy; immunopathogenesis; pharmacogenomics.