Researchers reverse diabetes in mice using AAV-mediated gene therapy

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Researchers at the University of Pittsburgh School of Medicine have achieved a sustained reversal of diabetes in mice using a gene therapy approach. The method used adeno-associated viral (AAV) vectors to deliver proteins which were able to reprogram alpha cells into functional beta cells which are destroyed by the immune system in patients with Type 1 diabetes.

The gene therapy induced the expression of two proteins, Pdx1 and MafA, in the pancreas of mice used in the study. Results showed successful reprogramming into beta cells, as well as normalization of blood glucose levels for an extended duration. The cells targeted for reprogramming are pancreatic alpha cells due to their location and abundance. The approach is able to bypass autoimmunity issues associated with beta replacement therapy by using cells that are initially distinct from the cells targeted by diabetes. The resultant cells appear to be insulin-producing, representing a viable replacement for the beta cells affected by the disease. The next stage of the research involves testing of the approach in primates before an application can be made to the FDA for trialing in humans. The study has been published in the journal Cell Stem Cell.

Senior author George Gittes stated, ‘This study is essentially the first description of a clinically translatable, simple single intervention in autoimmune diabetes that leads to normal blood sugars, and importantly with no immunosuppression. A clinical trial in both type 1 and type 2 diabetics in the immediate foreseeable future is quite realistic, given the impressive nature of the reversal of the diabetes, along with the feasibility in patients to do AAV gene therapy.’

Source: Gene therapy restores normal blood glucose levels in mice with type 1 diabetes. Press Release