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Diabetes Respite

Gene therapy was able to treat mice with type 1 diabetes for a short while, the Los Angeles Times reports.

A University of Pittsburgh-led team of researchers introduced viruses containing Pdx1 and MafA expression cassettes into the pancreatic ducts of mouse models of type 1 diabetes. Pdx1 is a transcription factor that enables pancreatic development, including beta cell maturation and proliferation, while MafA is a transcription factor that binds the insulin promoter to regulate its expression. By introducing these genes, the researchers hoped to reprogram alpha cells into insulin-producing beta cells.

As Pittsburgh's George Gittes and his colleagues report in Cell Stem Cell, treating mice in this way enabled them to produce insulin for about four months before returning to their original diabetic state.

He and his team say that this approach could potentially be used in humans. "The protection from recurrent diabetes in the mice was not permanent, although some studies would suggest that processes in mice are highly accelerated, so four months in mice might translate to several years in humans," Gittes says in a statement.

He further tells the LA Times that human clinical trials could be launched in the near future.