Current strategies and perspectives in insulin gene therapy for diabetes

Abstract

Insulin gene therapy is an approach that might overcome the weakness of islet cell therapy owing to its vulnerability to autoimmune attack. There are several mandatory conditions for successful insulin gene therapy. Efficient insulin gene therapy should have an effective insulin gene delivery mechanism, a system of regulation of the insulin biosynthesis that responds to glucose within extremely narrow physiological limits, a system of insulin processing into its active form and a choice of appropriate target cells, which possess biochemical characteristics similar to β cells, but are not targets for β-cell-specific self-reactivity. In this article, advantages and disadvantages of non-β-cell types that are most likely to be used for generating surrogate insulin-producing β cells are compared. Current achievements in insulin gene therapy are critically evaluated and future challenges are discussed.