Abstract

    Type I diabetes mellitus (T1DM) affects more than 17 million people worldwide, and current treatments for T1DM, known as insulin therapy, require continued insulin injection, diet control, and constant monitoring of blood sugar. Gene therapy methods that restore insulin production in non-pancreatic cells might provide a one-shot cure for T1DB. We propose a gene therapy for T1DM using an adeno-associated viral (AAV) vector that transfects somatic cells with an insulin gene controlled by a glucose-sensitive promoter, thus potentially restoring glucose-regulated insulin production in diabetic patients. Preliminary tests are conducted on cell lines to assess the efficiency of the therapy.

Background