Gene therapies and their potential application in Type 1 Diabetes

Abstract

Type 1 diabetes (T1D) is an autoimmune metabolic disorder, that arises due to autoimmune destruction of the β-cells in the islets of Langerhans in the pancreas. As a result, there is insufficient insulin production and ineffective glucose uptake, resulting in hyperglycemia. Currently available therapies are the widely used insulin injections and the not so common pancreatic islet transplantation. Even though, Insulin has been able to increase the life expectancy of T1D individuals, it is still shorter than the healthy population and the need for chronic uptake of immunosuppressive drugs, as well as the limited supply of healthy pancreatic islet cells that are needed for the islet transplantation, are some of the limiting factors of these two therapies. Therefore, scientists have been interested in developing other, more permanent treatments. Gene therapy has emerged as a potential therapeutic alternative to treat patients with T1D, even though its safety has not yet been established in humans. This thesis will focus on explaining the basics of gene therapy, as well as presenting the current status and future perspectives through analysis of various in vitro and in vivo studies, these include overexpression of genes or proteins involved in the molecular pathways leading to T1D.