Gene Therapy for Type 1 Diabetes Moves a Step Closer to Reality

  1. Timothy O’Brien
  1. Regenerative Medicine Institute and Department of Medicine, National Centre for Biomedical Engineering Science, National University of Ireland and Galway University Hospital, Galway, Ireland
  1. Corresponding author: Timothy O’Brien, timothy.obrien{at}

The prevalence of diabetes mellitus (DM) is assuming pandemic proportions and is currently estimated at 285 million cases (1). Although most cases are due to obesity-associated type 2 DM, there is also an increase in the annual prevalence of type 1 DM (2). It is estimated that 10% of the diabetic population have type 1 DM. Both forms of DM are associated with a long-term risk of microvascular and macrovascular complications (3) and the immediate risk of hypoglycemia.

There is abundant evidence that attainment of near normoglycemia will reduce the risk of complications associated with DM (4). However, attainment of near normoglycemia in patients with type 1 DM is limited by the occurrence of hypoglycemia. Patients with hypoglycemic unawareness are particularly prone to this problem as a limiting factor for achieving the required glycemic control. DM clinics globally have many patients with type 1 DM in whom recurrent hypoglycemia and the phenomenon of hypoglycemic unawareness present major clinical problems. Fortunately, there are many promising and exciting advances on the horizon for patients with this problem, including gene therapy as reported in this issue of Diabetes by Bosch and colleagues (5).

In the current study, the authors use an adeno-associated viral (AAV) vector to overexpress the genes for insulin and glucokinase in skeletal muscle in a canine model of DM. The study addresses the question of …

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