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Gene-based therapy for Type 1 diabetes mellitus: viral and nonviral vectors

Neha Shrestha, Francisca Araujo, Bruno Sarmento, Jouni Hirvonen & Helder A Santos

Type 1 diabetes mellitus (T1DM) is a global epidemic with an alarming number of patients suffering from the disease worldwide. The main goal of the therapy directed towards diabetes mellitus DM is achieving normal blood glucose levels. Gene therapy is one of the many approaches utilized to accomplish this in a safe, specific and efficient manner. Since the first cloning of insulin gene, the gene therapy approach for the treatment of T1DM has been widely investigated. Gene therapy for T1DM encompasses a broad area, from targeting noninsulin producing cells (e.g., hepatocytes, fibroblasts and myocytes) for stimulating the production and storage of insulin, and to genetically manipulate β cells to avoid autoimmune destruction. For this purpose, different types of viral and nonviral gene delivery vectors have been studied. This review aims mainly to discuss the most commonly gene delivery systems used for the treatment or prevention of T1DM. Several recent examples of viral and nonviral gene delivery vectors are provided, while pointing out the most relevant references found in the literature.