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Therapeutic Approaches for the Management of Autoimmune Disorders via Gene Therapy: Prospects, Challenges, and Opportunities


Rakesh K. Sindhu*, Piyush Madaan, Parteek Chandel, Rokeya Akter, G. Adilakshmi and Md. Habibur Rahman  


Background: Autoimmune diseases are the diseases that result due to the overactive immune response, and comprise systemic autoimmune diseases like rheumatoid arthritis (RA), sjӧgren’s syndrome (SS), and organ-specific autoimmune diseases like type-1 diabetes mellitus (T1DM), myasthenia gravis (MG), and inflammatory bowel disease (IBD). Currently, there is no long-term cure; but, several treatments exist which retard the evolution of the disease, embracing gene therapy, which has been scrutinized to hold immense aptitude for the management of autoimmune diseases.

Objective: The review highlights the pathogenic mechanisms and genes liable for the development of autoimmune diseases, namely T1DM, type-2 diabetes mellitus (T2DM), RA, SS, IBD, and MG. Furthermore, the review focuses on investigating the outcomes of delivering the corrective genes with their specific viral vectors in various animal models experiencing these diseases to determine the effectiveness of gene therapy.

Methods: Numerous review and research articles emphasizing the tremendous potential of gene therapy in the management of autoimmune diseases were procured from PubMed, MEDLINE, Frontier, and other databases and thoroughly studied for writing this review article.

Results: The various animal models that experienced treatment with gene therapy have displayed regulation in the levels of proinflammatory cytokines, infiltration of lymphocytes, manifestations associated with autoimmune diseases, and maintained equilibrium in the immune response, thereby hinder the progression of autoimmune diseases.

Conclusion: Gene therapy has revealed prodigious aptitude in the management of autoimmune diseases in various animal studies, but further investigation is essential to combat the limitations associated with it and before employing it on humans.


Immune system, autoimmune diseases, gene therapy, rheumatoid arthritis, type-1 diabetes mellitus, inflammatory bowel disease


Chitkara College of Pharmacy, Chitkara University, Punjab, Chitkara College of Pharmacy, Chitkara University, Punjab, Chitkara College of Pharmacy, Chitkara University, Punjab, Department of Pharmacy, Jagannath University, Sadarghat, Dhaka-1100, Department of PhysicxVikramaSimahpuri University, P.G. Centre, kavil-524201, Andhra Pradesh, Department of Pharmacy, Southeast University, Banani, Dhaka-1213

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