Japanese research suggests diabetes gene therapy potential

There are several ways to restore normal blood sugar levels in diabetics, including administration of insulin or pancreas and islet transplantation. However, the former involves daily injections and the latter requires life-long immunosuppressive therapy and is limited by tissue supply.

An alternative way to increase the amount of insulin circulating in the body is to enhance insulin gene transcription, which in turn results in an increase in the production of insulin. One possible way to do this is by increasing the body’s production of transcription factors, the molecules that are in charge of turning gene transcription on and off.

The Osaka University Graduate School of Medicine scientists used gene therapy to deliver three insulin transcription factors, MafA, PDX-1, and NeuroD, to the livers of diabetic mice. The transcription factors were inserted into an adenovirus and then the adenovirus was injected into the cervical vein of the mice. As a result, the mice experienced an increase in insulin gene expression and insulin production.

The researchers also discovered that overexpression of the three transcription factors in the livers of diabetic mice dramatically ameliorated glucose tolerance.

Although this technique has been successful in mice, adenovirus cannot be used to deliver genes into humans. Thus, it will be necessary to modify the vector or to develop some other technique to deliver the transcription factor genes into humans.