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The search for the new diabetes treatments has been an ongoing battle in science and medicine for many years. A number of novel strategies have been proposed in the rise of gene therapies, including insulin gene therapy as replacement for daily injections with monthly treatments, or complicated therapies targeting the immune system. However, these approaches are still far from a cure, merely providing a long-term treatment plan instead.

New strategies are being developed constantly, and an intriguing new study demonstrates that type-1 diabetes could be permanently reversed by a targeted gene therapy.

Type-1 diabetes mellitus is defined by the destruction of β-cells within pancreatic islets of Langerhans. Once these insulin-producing cells are destroyed, diabetes patients are faced with chronic hyperglycemia and are required to administer synthetic insulin several times a day. The latest research shows that neogenesis of β-cells from α-cells is possible by inactivation of a single transcription factor gene – Arx.

Upon inactivation, α-cells are shown to change their morphology completely and differentiate into functional insulin-producing β-cells. Diabetic mice return to normal glucose homeostasis after going through gene therapy and show an increased mass of pancreatic β-cells. The same rules are valid for different age cells, suggesting there is no age limit for β-cell regeneration. Even more importantly, hyperglycemia is reversed as effectively in drug-induced diabetic mice, which means this could become a universal therapy for different types of the disease.

While most gene therapies for diabetes concentrate on temporary treatments or target a sensitive immune system, the Arx-inactivation-driven β-cell neogenesis could provide a straight-forward and universal tool for treating a range of diabetic disorders. There is a hope not only for a short-term fix, but a permanent cure for diabetes, even though it is still a long way to develop a functional gene therapy for humans.

Source: www.technology.org