Diabetic nephropathy is the main cause of end-stage renal disease requiring dialysis in developed countries. This was compared with the direct injection of the vector into the portal circulation. Diabetes was induced in mice by i.p. The development of type 1 diabetes results from the almost total destruction of insulin-producing pancreatic β cells by autoimmune responses specific to β cells2,3,4,5,6. In healthy animals, insulin causes the glucose levels to return to normal in 90 minutes, while in diabetic animals carrying the SIA gene the glucose levels took 120 minutes to reach the same point. Both hepatic Ex4 RNA and plasma Ex4 were detectable at the end of the study. The liver subsequently went on to produce insulin.
These results suggest that mutant GCK may be a future cure for diabetes. Disorders arising from one gene mutation are the most promising candidates for gene therapy but a lot of the most common disorders such as diabetes, heart disease and arthritis