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

Rakesh K Sindhu; Piyush Madaan; Parteek Chandel; Rokeya Akter; Griddaluri Adilakshmi; Md Habibur Rahman

doi:10.2174/1566523221666210916113609

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

Curr Gene Ther. 2022;22(3):245-261. doi: 10.2174/1566523221666210916113609.

Authors

Rakesh K Sindhu¹, Piyush Madaan¹, Parteek Chandel¹, Rokeya Akter², Griddaluri Adilakshmi³, Md Habibur Rahman⁴

Affiliations

¹ Chitkara College of Pharmacy, Chitkara University, Punjab, India.
² Department of Pharmacy, Jagannath University, Sadarghat, Dhaka-1100, Bangladesh.
³ Department of Physicx, VikramaSimahpuri University, P.G. Centre, Kavil- 524201, Andhra Pradesh, India.
⁴ Department of Pharmacy, Southeast University, Banani, Dhaka-1213, Bangladesh.

PMID: 34530709
DOI: 10.2174/1566523221666210916113609

Abstract

Background: Autoimmune diseases are the diseases that result due to the overactive immune response, and comprehend 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 compete with 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.

Keywords: Immune system; autoimmune diseases; gene therapy; inflammatory bowel disease; rheumatoid arthritis; type-1 diabetes mellitus.

Publication types

Review

MeSH terms

Animals
Arthritis, Rheumatoid* / therapy
Autoimmune Diseases* / genetics
Autoimmune Diseases* / therapy
Diabetes Mellitus, Type 1* / therapy
Genetic Therapy
Inflammatory Bowel Diseases* / therapy