Diabetes "Cured" In Mice With Virus Therapy
phlack writes "Scientists at Baylor College of Medicine have found a way to treat diabetes in mice by using a virus (with the harmful genes removed) to trick the liver into working as a pancreas. This is still a ways away from working in humans, but it's progress, at least. Info can be found at Guardian and Science Daily."
FYI - The real question about this, and other gene therapy experiments, focueses on the safety of the vector being used. In this case an adenovirus virus was used. The virus itself is no longer virulent, but how does the target genetic material get integrated into the hosts genome? If it occurs at a specific site, then safety is maximized. If it occurs randomly, then you run the risk of knocking out genes where only a single healthy allele exists (loss of heterozygosity) and potentially, cancer.
Gene therapy holds a lot of promise, but the early cases of leukemia (remember the bubble boy cure? Two 'cured' patients subsequently developed cancer) make it prohibative. I'm an expert enough to know this a problem (in theory and in practice) but not enough of one to know how close we are to solving it.
-Sean
I just read the Nature Medicine article and the authors speculate that they were able to induce differentiation of hepatic stem cells or hepatocytes into islet-like cells, and it looks very convincing. A potential major shortcoming of this approach is not addressed, which is that in type I ("juvenile") diabetes, the islet cells are destroyed by an autoimmune response. Thus if you generate new self "pseudo islets", you may have present the very antigens that led to their destruction in the first place. The reason that is not a problem in this experiment is that the authors artificially destroy the islets with the toxin streptozotocin. The real test would be in an animal model that mimics type I diabetes, like the non-obese diabetic (NOD) mouse. I hope and assume that is the next critical experiment.