Gene Therapy: New Therapeutic approach to Diabetes Mellitus

Shilpa S. Borkar1*, Seema B. Wakodkar1, Pradeep S. Raghatate2, Debarshi Kar Mahapatra3

1Kamla Nehru College of Pharmacy, Butibori, Nagpur-441108, Maharashtra, India

2Department of Pharmaceutical Chemistry Kamla Nehru College of Pharmacy, Butibori, Nagpur-441108, Maharashtra, India

3Department of Pharmaceutical Chemistry, Dadasaheb Balpande College of Pharmacy, Nagpur-440037, Maharashtra, India

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In the past decade there has been a great deal of enthusiasm and high expectations for cell transplantation and genetic engineering. Type 1 diabetes mellitus (T1DM) is an autoimmune disorder characterized by T cell-mediated self-destruction of insulin-secreting islet β cells. Management of T1DM is challenging and complicated especially with conventional medications. One of the potential therapeutic alternatives to treat T1DM is emerged with Gene therapy. This review primarily focuses on the current status and the future perspectives of gene therapy in the management of T1DM. A number of the studies which are reported on gene therapy for the management of T1DM are performed in animal models and in preclinical studies. In addition, the safety of such therapies is yet to be established in humans. Currently, there are several gene level interventions and options that are being investigated, notably, would be the overexpression of genes and proteins needed against T1DM, transplantation of cells that express the genes against T1DM, stem-cells mediated gene therapy, genetic vaccination, immunological precursor cell-mediated gene therapy and vectors.

Keywords: Autoimmune disease, Gene therapy, Insulin, Type 1 diabetes mellitus, Proteins

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